Q2 2024 Sangamo Therapeutics Inc Earnings Call

Operator: Good afternoon, and welcome to the Sangamo Therapeutics second quarter 2024 teleconference call. Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker today, Louise Wilkie, Vice President of Investor Relations and Corporate Communications. Please go ahead.

Operator: Good afternoon and welcome to the Sangamo Therapeutics second quarter, 2024 teleconference call. Please be advised that today's conference is being recorded.

Good afternoon, and welcome to the Sangamo Therapeutics second quarter 2024 teleconference call.

Please be advised that today's conference is being recorded.

Louise Wilkie: I would now want to turn the conference over to your speaker today, Louise Wilkie, Vice President of Investor Relations and Corporate Communications. Please go ahead.

I'd now like to turn the conference over to your Speaker today, we use Wilkie Vice President Investor Relations and corporate Communications. Please go ahead.

Operator: Thank you.

Louise Wilkie: Thank you. Good afternoon, everyone. Thank you for joining us on the call today. On this call are several members of the Sangamo Executive Leadership Team, including Sandy Macrae, Chief Executive Officer, Prathyusha Duraibabu, Chief Financial Officer, Nathalie Dubois-Stringfellow, Chief Development Officer, Amy Pooler, Head of Research, and Greg Davis, Head of Technology. Slides from our corporate presentation can be found on our website, sangamo.com, under the Presentations page of the Investors and Media section.

Thank you good afternoon, everyone. Thank you for joining us on the call today.

Louise Wilkie: Good afternoon, everyone. Thank you for joining us on the call today.

Louise Wilkie: On this call, several members of the Sangamo Executive Leadership Team, including Sandy Macrae, Chief Executive Officer, Joshua Duraibabu, Chief Financial Officer, Nathalie Dubois Duraibabu, Chief Development Officer, Amy Pooler, Head of Research, and Greg Davis, Head of Technology. Slides from our corporate presentation can be found on our website, Sangamo.com, under the presentations page of the Investors and Media section.

Speaker Change: On this call are separate members of the Sangamo executive leadership team, including Sandy Macrae, Chief Executive Officer, She should do or Bobby Chief Financial Officer knocked leads your Barstools delight, Chief Development Officer, Amy pool, our head of research and Greg Davis head of technology slots.

Speaker Change: Slides from our corporate presentation can be found on our website.

Speaker Change: And presentations page of the investors and media section.

Louise Wilkie: This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements relating to Sangamo's cash runway, plans to obtain additional capital, and ability to continue to operate as a going concern, the therapeutic and commercial potential of Sangamo's product candidates and technologies, Sangamo's ability to earn and receive payments from its collaboration on license agreements, including the Genentech and Pfizer agreements, Sangamo's expectations regarding new collaboration and license agreements, the anticipated plans and timelines of Sangamo and its collaborators for clinical trials, clinical data presentations and regulatory submissions, upcoming catalysts and milestones, and other statements that are not historical facts.

Louise Wilkie: This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements relating to Sangamo's cash runway, plans to obtain additional capital, and ability to continue to operate as a going concern. The therapeutic and commercial potential of Sangamo's product candidates and technologies, Sango has the ability to earn and receive payments from its collaboration and license agreements, including the Genente Conviser Agreement. So as sorry, Sangamo's expectations regarding new collaboration and license agreements, the anticipated plans and timelines of Sangamo and its collaborators for clinical trials, clinical data presentations and regulatory submissions, upcoming catalysts and milestones, and other statements as a not historical fact.

Speaker Change: This call includes forward looking statements regarding Sangamo current expectations. These statements include but are not limited to statements relating to fund with cash on my plans to obtain additional capital and ability to continue to operate as a going concern the therapeutic and commercial potential of <unk> product candidates and technologies.

Speaker Change: Its ability to Ana receive payments from our collaboration and license agreements and placing them with genentech and Pfizer agreements.

Speaker Change: I'm, sorry, sangamo expectations regarding new collaboration and license agreements.

Speaker Change: Despite the plans and timelines of Sangamo and its collaborators for clinical trials clinical data presentations and regulatory submissions.

Speaker Change: From a catalysts and milestones and other statements that are not historical fact actual.

Louise Wilkie: Actual results may differ from what we discussed today. These statements are subject to certain risks and uncertainties of the discussed and are filing for the SEC. Specifically on an annual report and Form 10-K for the fiscal year ended December 31, 2023, supplemented by Sangamo's quarterly reports on Form 10-Q for the quarters ended March 31, 2024, and June 30, 2024.

Louise Wilkie: However, actual results may differ materially from what we discussed today. These statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC, specifically in our annual report on Form 10-K for the fiscal year ended December 31, 2023, as supplemented by Sangamo's quarterly reports on Form 10-Q for the quarters ended March 31, 2024, and June 30, 2024, and subsequent filings and reports that Sangamo makes from time to time with the SEC.

Speaker Change: Actual results may differ materially from what we discuss today.

Speaker Change: The statements are subject to certain risks and uncertainties are discussed in our filings with the SEC specifically on our annual report on Form 10-K for the fiscal year ended December 31, 2023 are supplemented by starting to my quarterly reports on Form 10-Q for the quarters ended March 31, 'twenty 'twenty four and June 30, 'twenty 'twenty four and subsequent filings.

Speaker Change: So think of it makes from time to time with the SEC.

Louise Wilkie: The forward-looking statements' data today are made out of today, and we're going to take no duty to update such information except as required by law. Please note that all forward-looking statements about our future plans and expectations are subject to our ability to secure adequate additional funding.

Louise Wilkie: The forward-looking statements stated today are made as of today, and we undertake no duty to update such information except as required by law. Please note that all forward-looking statements about our future plans and expectations are subject to our ability to secure adequate additional funding. Now, I'll turn the call over to our CEO, Sandy Macrae.

Speaker Change: The forward looking statements stated today are made as of today and we want to take no duty to update such information, except as required by law.

Speaker Change: All forward looking statements about our future plans and expectations are subject to our ability to secure adequate additional funding.

Sandy Macrae: Now, I'll turn the call over to our CEO, Sandy McCray. Thank you, Louise, and good afternoon to everyone joining the call today. This has been such an exciting and important quarter for Sangamo. At our last earnings call, we shared compelling pre-clinical data demonstrated the potential of our neurology epigenetic regulators, AAV capsid delivery platform, and next generation genome engineering platform. Importantly, we also outlined a resolute focus on raising additional capital in order to progress these compelling assets into the clinic and supports patients in need.

Speaker Change: Now I'll turn the call over Fussier Sandy Macrae.

Sandy Macrae: Thank you, Louise, and good afternoon to everyone joining the call today. This has been such an exciting and important quarter for Sangamo. At our last earnings call, we shared compelling preclinical data demonstrating the potential of our neurology epigenetic regulators, AAV capsid delivery platform, and next generation genome engineering platform. Importantly, we also outlined our resolute focus on raising additional capital in order to progress these compelling assets into the clinic and towards patients in need.

Sandy Macrae: Thank you Luis and good afternoon to everyone joining the call today.

Sandy Macrae: This is being such an exciting and important quarter for <unk>.

Sandy Macrae: I've heard last earnings call, we shared compelling preclinical data demonstrating the potential of our neurology epigenetic regulators AAV capsid delivery platform.

Sandy Macrae: Next generation genome engineering platform.

Sandy Macrae: Importantly, we also find a resolute focus on raising additional capital in order to progress these compelling assets into the clinic and supports patients in need.

Sandy Macrae: To that end, this morning we were thrilled to announce our first Neurology Epigenetic Regulator and Delivery Capsid Licence Agreement. This comes close on the heels of the highly compelling Phase 3 affine data for the haemophilia A gene therapy we're co-developing with Pfizer. Both announcements demonstrate external interest in and validation of the potential of our science, and both could meaningfully extend our cash runway as we continue to advance our wholly-owned neurology epigenetic regulation pipeline.

Sandy Macrae: To that end, this morning we were thrilled to announce our first neurology epigenetic regulator and delivery capsid license agreement. This comes close on the heels of the highly compelling phase 3-year fine data for the hemophilia ageing therapy we are co-developing with Pfizer. Both announcement stems straight external interest in and validation of the potential of our signs, and both quick meaningfully extend our cash runway as we continue to advance our wholly-owned neurology epigenetic regulation pipeline. Getting into the details of our STAC BBB CAPTED deal, this morning we announced a license agreement with Genentech to develop novel, intravenously administered genomic medicines for neurodegenerative disease.

Sandy Macrae: To that end. This morning, we were thrilled to announce our first neurology epigenetic regulator and delivery capsid license agreement.

Sandy Macrae: This comes close on the heels of the highly compelling phase III you find data for the hemophilia a gene therapy, we're co developing with Pfizer.

Sandy Macrae: Both announcements demonstrate external interest in and validation of the potential of our science and both could meaningfully extend our cash runway as we continue to advance our wholly owned neurology epigenetic regulation pipeline.

Sandy Macrae: Getting into the details of our STAC BBB capsid deal, this morning we announced a license agreement with Genentech to develop novel intravenously administered genomic medicines for neurodegenerative disease. We have granted Genentech an exclusive license to our highly potent zinc finger repressors that are targeted to tau, a critical gene involved in Alzheimer's disease and other tauopathies, as well as an additional undisclosed second neurology target.

Sandy Macrae: Getting into the details of our stack BBB capsid deal. This morning, we announced a license agreement with Genentech to develop novel intravenously administered genomic medicines for neuro degenerative disease.

Sandy Macrae: We have granted Genentech an exclusive license to our highly potent zinc finger repressors that are targeted to tau, a critical gene involved in Alzheimer's disease and other tauopathies, as well as an additional, undisclosed second neurology target. For these same targets, we've also granted Genentech an exclusive license to our industry-leading Neurotropic Delivery Capsid STAC BBB, which has demonstrated potent blood-brain barrier penetration and brain transduction in non-human primates. As we have shared before, we strongly believe that our powerful combination of proprietary genome targeting cargo paired with our Neurotropic Delivery Capsid platform is critical to the successful development of neurology-focused genomic medicines.

Sandy Macrae: We have granted genentech, an exclusive license to a highly potent zinc finger repressor, they're targeted to tout a critical gene involved in Alzheimer's disease, another tie all parties.

Sandy Macrae: That's what I listen additional undisclosed second neurology target.

Sandy Macrae: For these same targets, we've also granted Genentech an exclusive license to our industry-leading neurotropic delivery capsid, STAC-BBB, which has demonstrated potent blood-brain barrier penetration and brain transduction in non-human primates. As we have shared before, we strongly believe that our powerful combination of proprietary genome targeting cargo paired with our neurotropic delivery capsid platform is critical to the successful development of neurology-focused genomic medicine. We believe this agreement with Genentech, a leader in the biotechnology and neurology space, reinforces this potent combination and underscores how Sangamo is able to provide both.

Sandy Macrae: For these same targets, we post who granted genentech, an exclusive license to our industry, leading neutral peak delivery capsid stack BBB.

Sandy Macrae: As demonstrated potent blood brain barrier penetration and brain transduction in nonhuman primates.

Sandy Macrae: As we have shared before we strongly believe that our powerful combination of proprietary genome targeting cargo paired with our neutral peak delivery capsid platform is critical to the successful development of neurology focused genomic medicines.

Sandy Macrae: We believe this agreement with Genentech, a leader in the biotechnology and neurology space, reinforces this potent combination and underscores how Sangamo is able to provide both. We are delighted that Genentech chose our zinc finger and capsid delivery technologies to address their neurology needs. We expect to receive from Genentech $50 million in near-term upfront license fees and mouse room payments, which we anticipate will extend our cash runway into early 2025 and are eligible to earn up to $1.9 billion in development and commercial mouse room payments, as well as tiered royalties or net sales of such products under the agreement.

Sandy Macrae: We believe this agreement with Genentech, a leader in the biotechnology and neurology space reinforces this potent competition underscores how sangamo is able to provide both.

Sandy Macrae: We are delighted that Genentech chose our SyncFinger and Capsid delivery technologies to address their neurology needs. We expect to receive from Genentech $50 million in near-term upfront license fees and milestone payments, which we anticipate will extend our cash runway into early 2025, and are eligible to earn up to $1.9 billion in development and commercial milestone payments, as well as tiered royalties or net sales of such products under the agreement. Our agreement with Genentech is significant as we believe it paves the way for others in the future.

Sandy Macrae: We are delighted that genentech chose our zinc finger capsid delivery technologies to address their neurology needs.

Sandy Macrae: We expect to receive from Genentech $50 million in near term upfront license fees and milestone payments, which we anticipate will extend our cash runway into early 2025.

Sandy Macrae: Eligible to earn up to $1 $9 billion in development and commercial milestone payments as well as tiered royalties on net sales of such products under the agreement.

Sandy Macrae: Our agreement with Genentech is significant as we believe it paves the way for others in the future. We anticipate this agreement could be the first of multiple capsid collaborations, as we have ongoing discussions with other interested parties, alongside our Fabri partnership discussions. Generating additional funding continues to be our top priority as we work to position Sangamo for long-term success in value creation, and are hopeful to have news of additional transactions in the second half of this year.

Sandy Macrae: IRA agreement with Genentech is significant as we believe it pays paves the way for others in the future.

Sandy Macrae: We anticipate this agreement could be the first of multiple CAHPSID collaborations as we have ongoing discussions with other interested parties alongside our FABRI partnership discussions. Generating additional funding continues to be our top priority as we work to position Sangamo for long-term success and value creation, and we are hopeful to have news of additional transactions in the second half of this year. I would now like to hand over to Nathalie, our head of development, who will share details on the positive top line phase three affine trial results and take us through other pipeline updates.

Sandy Macrae: We anticipate disagreement could be the first stroke multiple caps and collaborations as we have ongoing discussions with other interested parties alongside our fabry partnership discussions.

Sandy Macrae: January thing additional funding continues to be our top priority as we work to position <unk> for long term success and value creation and are hopeful to have news of additional transactions in the second half of this year.

Nathalie Dubois: I would now like to hand over to Natalie, our head of development, who will share details on the positive top-line phase three affine trial results and take us through other pipeline updates. Natalie.

Sandy Macrae: I would now like to hand over to Natalie our head of development, who will share details from the positive top line phase III <unk> trial results and take us through other pipeline updates.

Nathalie Dubois: Thank you, Sandy. As Sandy outlined, we were thrilled to recently share positive top line results from the phase three Affine trial of Europe Tokaji in Fittel-Farbervek, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia A. Hopefully, we had an opportunity to look at the results, but to summarize the key point, the Affine trial achieves its primary objective of non-inferiority as well as superiority of total annualized bleeding rate or ABR, compared with routine factor rate replacement prophylaxis streaks. Following a single dose of Giro Taka-Jean, Fetal-Pavo-Vex demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period.

Sandy Macrae: Lee.

Nathalie Dubois-Stringfellow: and Sandy. As Sandy outlined, we were thrilled to recently share positive top-line results from the Phase III affine trial of Gerotocogene Fetal Fibroblast, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia. Hopefully, we had an opportunity to look at the results. But to summarize the key points,

Natalie: Thank you Sandy.

Natalie: Sandy outlined we were thrilled to recently share positive topline results from the phase III assign trial.

Lee: You took the GNC telework and investigational gene therapy, we are developing with Pfizer for patient with moderately severe to severe hemophilia a.

Speaker Change: Hopefully <unk> had an opportunity to look at the results, but to summarize the key point.

Nathalie Dubois-Stringfellow: The AFIND trial achieved its primary objective of non-inferiority, as well as superiority of total annualized bleeding rate, or ABR, compared with routine factor VIII replacement prophylaxis treatment following a single dose of factor VIII. Giro Takuchi and Fidel Pavlovic demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period. G-secondary endpoints were met and also demonstrated superiority compared to prophylaxis, with 84% of participants maintaining factor VIII activity greater than 5% at 15 months post-infusion, and the majority having factor VIII activity greater than or equal to 15%. Furthermore, the mean treated ABR showed a 98.3% reduction post infusion. Importantly, the product candidate was generally well-tolerated.

Speaker Change: Fine trial achieved its primary objective of non inferiority and superiority of total annualized bleeding rate or ABR compare with routine factor replacement prophylaxis treatment.

Speaker Change: Following a single dose.

Speaker Change: Taco GNC tell Pablo <unk> demonstrated a statistically significant reduction in mean total ABR compared to the premium features period.

Speaker Change: Key secondary endpoints were met and also demonstrated superiority compared to prophylaxis with 84% of participant maintaining fluctuate activity growth greater than 5% at 15 months post infusion and the majority are <unk>.

Nathalie Dubois: Having factorate activity greater than or equal to 15%. Furthermore, the mean treated ABR showed a 98.3% reduction post-infusion. Importantly, the product candidate was generally well-paralleled.

Speaker Change: <unk> fluctuate activity greater than or equal to 15%.

Speaker Change: Furthermore, the men treated ABR showed a 98, 3% reduction post infusion.

Speaker Change: Fortunately the product candidate was generally well tolerated.

Nathalie Dubois: This impressive result further validated the potential of our genomic technologies and take us one step closer towards what could become Sangamo's first medicine commercially available to patients. We greatly appreciate Pfizer's strong leadership of this important program, and we are therefore pleased to see this update prominently profile in their second quarter earnings update last week. Pfizer reiterated that they plan to review this data with regulatory authorities in the coming months, which would take us a further step closer to unlocking a substantial first tranche of the anticipated milestone payments. As a reminder, we are eligible to earn up to $220 million in milestone payment from Pfizer upon the achievement of certain regulatory and commercial milestones and 14 to 20% royalty on potential sales for this program if approved and commercialized.

Nathalie Dubois-Stringfellow: These impressive results further validate the potential of our genomic technologies and take us one step closer to what could become Sangamo's first medicine commercially available to patients. We greatly appreciate Pfizer's strong leadership of this important program, and we're therefore pleased to see this update permanently profiled in their second quarter earnings update last. Pfizer reiterated that they plan to review this data with regulatory authorities in the coming months, which would take us a further step closer to unlocking a substantial first tranche of the anticipated milestone payment.

Speaker Change: Impressive results further validate the potential of our genomic technologies and take US one step closer towards what could become Sangamo first medicine commercially available to patients.

Speaker Change: We greatly appreciate the strong leadership of this important program and we are therefore pleased to see this update permanently profile in the second quarter earnings update last week.

Speaker Change: Pfizer reiterated that they plan to review these data with regulatory authorities in the coming months, which would take US a first step closer to unlocking a substantial first tranche of the anticipated milestone payments.

Nathalie Dubois-Stringfellow: As a reminder, we are eligible to earn up to $220 million in milestone payments from Pfizer upon the achievement of certain regulatory and commercial milestones and 14% to 20% royalty on potential sales for this program if approved and commercialized.

Speaker Change: As a reminder, we are eligible to earn up to $220 million in milestone payments from Pfizer upon the achievement of certain regulatory and commercial milestones.

Speaker Change: And 14% to 20% royalty on potential sales for this program if approved and commercialized.

Nathalie Dubois: Positively impacting the lives of patients is our ultimate goal, so selecting a partner with strong commercialization infrastructure and experience, as well as a broader franchise in this area, was an essential criterion when selecting a partner for this program some years ago. We look forward to hearing Pfizer's progress on this program in the coming months.

Nathalie Dubois-Stringfellow: Positively impacting the lives of patients is our ultimate goal, so selecting a partner with strong commercialization infrastructure and experience, as well as a broader franchise in this area, was an essential criterion when selecting a partner for this program some years ago. We look forward to hearing Pfizer's progress on this program in the coming months. This quarter, we also continue to advance our Fabry disease program, and I wanted to take a moment to share how the Fabry clinical data continues to evolve and demonstrate real patient benefits.

Speaker Change: Positively impacting the lives of patients is our ultimate goal, so selecting a partner with strong commercialization infrastructure and experience as well as the broader franchise in this area with an essential criterion when selecting a partner for this program some years ago.

Speaker Change: We look forward to hearing Pfizer's progress on this program in the coming months.

Nathalie Dubois: This quarter, we also continue to advance our Fabry disease program, and I wanted to take a moment to share how the Fabry clinical data continues to evolve and demonstrate real patient benefits, as outlined previously. Dozing is complete in the Phase 1, 2 star study of Isaraga gene Siva Parvavec and investigational gene therapy for the treatment of Fabry disease with a total of 33 patient dose. Since our last update, we are pleased that three additional patients have been able to stop enzyme replacement therapy or ERT, resulting in a total of 17 patients withdrawn from ERTs to date.

Speaker Change: This quarter. We also continued to advance our fabry disease program and I wanted to take a moment to share how the fabry clinical data continues to evolve and demonstrate real patient benefit.

Nathalie Dubois-Stringfellow: As outlined previously, dosing is complete in the Phase 1, 2 star study of Eseragaji and Sivaparvava and the Investigational Gene Therapy for the Treatment of Fabry Disease with a total of 33 patient deaths. Since our last update, we are pleased that three additional patients have been able to stop enzyme replacement therapy, or ERT, resulting in a total of 17 patients withdrawn from ERT to date. All 17 patients remain off ERT as of today.

Speaker Change: As outlined previously dosing is complete in the phase one to start a study of.

Speaker Change: Is around the TNC about pause of BEC and investigational gene therapy for the treatment of Fabry disease with a total of 33 patients dosed.

Speaker Change: Since our last update we are pleased that the three additional patients have been able to stop enzyme replacement therapy or E. R. T, resulting in a total of 17 patients withdrawn from <unk> to date.

Nathalie Dubois: All 17 patients remain off ERT as of today. The one remaining patient dose who began the study on ERT has plans in place to withdraw ERT treatment at the appropriate time to reiterate. 17 patients no longer have to undergo long ERT infusion sessions every second week. This is a life-changing development for those patients. With the longest treated patient now to nearly four years and with 10 patients having at least two years of full up, we continue to immerse important clinical data, including durability from this study. This data continued to look highly encouraging, with patient achieving and maintaining physiological or super physiological level of plasma alpha-gal A enzyme activity.

Speaker Change: 17 patients remain our CRT as of today. The one remaining patient dose will begin to study on the <unk> as planned to place two weeks to IRT treatment at the appropriate time.

Nathalie Dubois-Stringfellow: The one remaining patient dosed will begin the study on ERT as planned in place to withdraw ERT treatment at the appropriate time. To reiterate, 17 patients no longer have to undergo a long ERT infusion session every second week. This is a life-changing development for those patients. With the longest-treated patient now at nearly four years, and with 10 patients having at least two years of follow-up, we continue to amass important clinical data, including durability, from this study.

Speaker Change: To reiterate 17 patients no longer have to undergo long E. R. T. Infusion session. Every second week. This is a life changing development for those patients.

Speaker Change: With the longest treated patient and know what to nearly four years and with temptation nothing at least two years of follow up we continue to amass important clinical data, including durability from this study.

Nathalie Dubois-Stringfellow: These data continue to look highly encouraging, with patients achieving and maintaining physiological or supraphysiological levels of plasma alpha-GalA enzyme activity. What particularly pleases me is that we are seeing evidence of improvement in kidney function. In contrast to the progressive decline in kidney function in untreated Fabry patients and even those on ART, in the 18 patients treated for more than one year, we are seeing a statistically significant rise in both mean and median EGFR levels in male and female patients, those with erysalgogy and sivoparvovac. This reflects an important improvement in kidney function. We look forward to sharing a detailed data update in the coming months.

Speaker Change: These data continue to look highly encouraging with patient achieving and maintaining physiological or super bossy theoretical level of plasma alpha Gal a enzyme activity.

Nathalie Dubois: What particularly pleases me is that we are seeing evidence of improvement in kidney function. In contrast to the progressive decline in kidney function seen in untreated fabrication and even those on ART, in the 18 patients treated for more than one year, we are seeing a statistically significant rise in both mean and median EGFR levels, in male and female patients, those with erasagogy and silver parvovac. This reflects an important improvement in kidney function.

Speaker Change: What particularly pleased to see is that we are seeing evidence of improvement in kidney function.

Speaker Change: Trust to the progressive decline in kidney function in untreated fabry patient and even those on air to heat in the 18 patients treated for more than one year. We are seeing a statistically significant rise in both mean and median egfr levels in male and female patients.

Speaker Change: With EBITA GNC, but part of it.

Speaker Change: This reflects an important improvement in kidney function, we look forward to sharing a detailed data update in the coming months.

Nathalie Dubois: We look forward to sharing a detailed data update in the coming months.

Nathalie Dubois: In June, we also held a productive meeting with the European Medicines Agency, or EMA, on a proposed pathway to potential approval in Europe. In particular, we were impressed to be joined in that discussion with nine members of the US Food and Drug Administration.

Nathalie Dubois-Stringfellow: In June, we also held a productive meeting with the European Medicine Agency, or EMA, on a proposed pathway to potential approval in Europe. In particular, we were impressed to be joined in that discussion by nine members of the U.S. Food and Drug Administration. Finally, our engagement with potential Fabry collaboration partners continues, with multiple discussions ongoing.

Speaker Change: In June we also held a productive meeting with the European Medicines agency or EMA on a proposed pathway to potential approval in Europe. In particular, we were impressed to be joining that discussion with nine member of the U S food and drug administration.

Nathalie Dubois: Finally, our engagement with potential fabric collaboration partners continues with multiple discussions ongoing. This advances allows us to focus on our mission as a genomic medicine company to treat debilitating neurological disorders. We believe our ability to combine potent zinc-finger epigenic regulation payloads with exciting new industry-leading capsid delivery technology could unlock significant potential for the treatment of devastating neurological diseases, indication for which delivery to the central neuro system has historically proved challenging. This quarter, we continue to advance IND-enabling activities for zinc-finger repressor in chronic neuropiting pain, not 1.7, as well as CTA-enabling activity for our program to treat prion disease, which leverages our novel STAC BBB capsid.

Speaker Change: Finally, our engagement with potential Fabry collaboration partners continues with multiple discussions ongoing.

Nathalie Dubois-Stringfellow: These advances allow us to focus on our mission as a genomic medicine company to treat debilitating neurological disorders. We believe our ability to combine potency finger epigenetic regulation payloads with exciting new industrially encapsulated delivery technology could unlock significant potential for the treatment of devastating neurological diseases, indications for which delivery to the central nervous system has historically proved challenging. This quarter, we continue to advance IND-enabling activities for our zinc finger repressor in chronic neuropathic pain.

Speaker Change: These advances allows us to focus on our mission as a genomic medicine company to treat debilitating neurological disorders, we believe our ability to combined put in zinc finger epigenetic regulation payloads with exciting new industry, leading capsid delivery technology could unlock significant.

Speaker Change: Potential for the treatment of <unk>.

Speaker Change: Saving neurological diseases.

Speaker Change: Indications for which delivery to the central nervous system has historically proved challenging.

Speaker Change: This quarter, we continue to advance our IND, enabling activities for our zinc finger repressor in chronic neuropathic pain.

Nathalie Dubois-Stringfellow: NAV1.7 as well as CTA enabling activity for our program to treat prion disease, which leverages our novel stack BBB cap. And we also shared exciting developments in our next generation genomic engineering efforts to integrate larger sequences of DNA into the genome, an approach that could offer the potential to treat with a single medicine patients who have unique mutations in the same gene. I will now hand it back to Sandy for her closing remarks. Thank you.

Speaker Change: One seven as well as Cta, enabling activity for our program to treat prion disease, which leverages our novel stack BBB capsid.

Nathalie Dubois: And we also shared exciting development in our next-generation genomic engineering efforts to integrate larger sequences of DNA into the genome, an approach that could offer the potential to treat with a single medicine patients who have unique petations in the same gene.

Speaker Change: And we also shared exciting development in our new next generation genomic engineering efforts to integrate largest sequences of DNA into the genome and approach that could offer the potential to treat with a single medicine patient who have unique mutation in the same team.

Sandy Macrae: I will now hand it back to Sandy for closing remarks. Thank you, Natalie.

Sandy Macrae: I will now hand, it back to sandy for closing remarks.

Sandy Macrae: Thank you Natalie.

Sandy Macrae: In closing, we're delighted with the important progress made this quarter, which can be summarised in three areas. First, we are proud that our epigenetic regulation and capsid delivery capabilities are being recognized by leaders in the field. We expect that the near-term payments to be received from Genentech will extend our cash runway into the first quarter of 2025, reinforcing the potential of our science and providing us with needed near-term financial resources. We believe this could be the first of multiple CAHPSID collaborations with other partners, which we hope will create a stream of future non-dilutive funding opportunities.

Sandy Macrae: In closing, we're delighted with the important progress made this quarter, which can be summarized in three areas. First, we are proud that our epigenetic regulation and capsid delivery capabilities are being recognized by leaders in the field. We expect that the near-term payments to be received from Genentech will extend our cash from way into the first quarter of 2025, reinforcing the potential of our science and providing us with needed near-term financial resources. We believe this could be the first of multiple capsid collaborations to come with other partners, which we hope will create a stream of future non-dilutive funding opportunities.

Sandy Macrae: In closing, we're delighted with the important progress made this quarter of which can be summarized in three areas.

Sandy Macrae: First we are proud that our epigenetic regulation capsid delivery capabilities are being recognized by leaders in the field.

Speaker Change: We expect that the near term payments to be received from Genentech will extend our cash runway into the first quarter of 2025, reinforcing the potential of our science and providing us with needed near term financial resources.

Sandy Macrae: We believe this could be the first of multiple capsid collaborations to come with other partners, which we hope will create a stream of future non dilutive funding opportunities.

Sandy Macrae: Second, we are excited to have achieved important advances in our legacy gene therapy programmes, which provide a foundation for our core neurology business. The positive top-line data from the Phase III Affine trial take us one step closer toward what could be Sangamo's first meds and commercially available to patients, and the resulting important potential milestones and royalties that would follow. The compelling clinical data emerging from our Fabry Disease Program, coupled with the encouraging potential regulatory pathway, further underscores the importance of our science and we anticipate will further fund the company.

Sandy Macrae: Second, we are excited to have achieved important advances in our legacy gene therapy programs, which provide a foundation for our core neurology business. The positive top-line data from the Phase 3 affine trial take us one step closer to what could be Sangamo's first medicine commercially available to patients, and the resulting important potential milestones and royalties that would follow. The compelling clinical data emerging from our Fabry disease program, coupled with the encouraging potential regulatory pathway, further underscores the importance of our science, and we anticipate will further fund the company.

Sandy Macrae: Second we are excited to have achieved important advances in our legacy gene therapy programs, which provides the foundation for our core neurology business.

Sandy Macrae: Positive topline data from the phase III <unk> trial take us one step closer towards could be Sangamo first medicine commercially available to patients and the resulting important potential milestones and royalties that would follow.

Sandy Macrae: The compelling clinical data emerging from our fabry disease program, coupled with encouraging potential regulatory pathway further underscores the importance of our science and we anticipate will further fund the company.

Sandy Macrae: Third, this stream of non-deleted funding opportunities provides the foundation to build our Neurology Epigenetic Regulation Pipeline and capsule delivered platform led by our chronic neuropathic pain and prime disease programs, both of which continue to advance towards potential IND and CTA submissions. We will continue to build our progress in the second half of 2024 and look forward to continuing to achieve key milestones to bring our promising technology to patients in need.

Sandy Macrae: Third, this stream of non-dilutive funding opportunities provides the foundation to build our neurology epigenetic regulation pipeline and CAPSEI delivery Platform, led by our Chronic Neuropathic Pain and Prone Disease Programs, both of which continue to advance towards potential IND and CTA submission. We will continue to build on our progress in the second half of 2024 and look forward to continuing to achieve key milestones to bring our promising technology to patients in need. Operator, please open the line for questions.

Speaker Change: Third this stream of non dilutive funding opportunities provides a foundation to build our neurology epigenetic regulation pipeline and KFC delivered.

Sandy Macrae: Platform led by our chronic neuropathic pain and prime disease programs, both of which continue to advance towards potential IND and Cta submissions.

Sandy Macrae: We will continue to build on our progress in the second half of 2024 and look forward to continuing to achieve key milestones to bring a promising technology to patients in need.

Operator: Operator, please open the line for questions. Thank you. At this time, we will conduct the question-and-answer session.

Speaker Change: Operator, please open the line for questions.

Operator: Thank you. At this time, we will conduct the question and answer session. As a reminder, to ask a question, you will need to press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again.

Speaker Change: Thank you at this time, we will conduct a question and answer session.

Operator: As a reminder, to ask a question, you will need to press star 11 on your telephone and wait for your name to be announced. To withdraw your question, please press star 11 again. Please stand by while we compile the Q&A roster.

Speaker Change: As a reminder to ask a question you will need to press star one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one again please.

Speaker Change: Please standby, while we compile the Q&A roster.

Nicole Germino: Our first question comes from the line of Nicole Germino. Through with securities, your line is now open. Great. Thanks so much for taking my question and for your question.

Speaker Change: Our first question comes from the line of Nicole Jeremy.

Nicole Jeremy: Sure with Securities. Your line is now open.

Nicole Jeremy: Great. Thanks, so much for taking my question and congrats on the progress.

King: King you've noted that BBB.

Speaker Change: BBB is manufacturable.

Speaker Change: Can you elaborate a bit more than what the police essentially from a cost perspective compared to process a compare to the standard.

Operator: Thank you for your question.

Speaker Change: Okay.

Amy: Thank you for your question Amy can you help with that please yeah sure I'm happy to comment on that once the discovery of stack Pvp was made one of the first things that we did just look into the manufacture ability of the of the capsid knowing that that's an important important consideration when scaling up in order to be able to make another.

Amy Pooler: Amy, can you help with that? Please.

Amy Pooler: Sure. I'm happy to comment on that.

Amy Pooler: Once the discovery of SACBBB was made, one of the first things that we did is look into the manufacturability of the capsid, knowing that that's an important consideration when scaling up in order to be able to make enough of the capsid to treat these devastating neurological diseases we mentioned earlier. What we found is that, by and large, the SACBBB capsid behaves very similarly to its parent capsid, so we believe that all of the expertise that we've developed for our previous programs and manufacturing AV can be leveraged now for SACBBB.

Sandy Macrae: Thanks, Amy.

unknown: Thanks, Amy. And it's one of the gating factors in

Sandy Macrae: And it's one of the gating factors in choosing this capsid to move ahead. Thank you.

Operator: One moment for our next question.

Gina Wang: Our next question comes from the line of Gina Wang of Barclays. Your line is now open. Thank you for taking my question.

Gina Wang: So I wanted to come where it's on the degree deal with genetic tech. So we wanted to ask regarding the partnership. They're open for additional indication beyond the two indications that already decided regarding, and then second regarding the February disease. Now, you have a more compelling clinical data and also strength and cash position. What is your latest thoughts on the February program?

Speaker Change: Regarding the steel.

Speaker Change: And then secondly regarding the fabry disease now you'll have a more compelling clinical data and also strengthened cash position what is your latest thoughts.

Speaker Change: On the Fabry program path forward, whether as a standalone asset we wanted to continue looking for the partner to divest this asset.

Sandy Macrae: Pass forward, whether as a standalone asset, or you want to continue looking for the partner to devise this asset?

Sandy Macrae: So you have to excuse us; you were very quiet. Can I make sure that we understood the question? So I think the first one was about Stack BBB and was it available for other indications? And would we be doing partnering for other indications? Did I understand that correctly? That's correct. Sorry. Yeah. Can you hear me better now? Yes, that's much better. Thank you. So the the license we have with or the partnership we have with Genentech is for the name their first indication is tau, which clearly applies to Alzheimer's and the various toe opathies and a second undescribed target.

Speaker Change: So you have to excuse US you were very quiet can I make sure that we understood. The question. So I think the first one was about stock BBB, mostly to available for other indications and we'd be doing partnering for other indications did I understand that correctly. That's correct sorry, yes can you hear me better.

Speaker Change: Yes, that's much better. Thank you so the <unk> the.

Speaker Change: The license, we have with <unk> or the partnership we have with Genentech is for the named the first indication is toe twitch.

Speaker Change: Clearly.

Speaker Change: Applies to Alzheimer's and the various type of disease and a second undisclosed target.

Sandy Macrae: All other targets are unclaimed at the moment. Of course, we at Sangamo want to take forward Prime, and we have a range of, I think, there's eight in our current pipeline on the website that we would consider, depending on resources, taking forward. We're really lucky that we've been inundated by other companies looking for opportunities to access the capsid, and we're being very careful to hear the ones that they're interested in and make sure that that fits with the ones that we want to take forward. So it really does speak to the importance of this capsid and the many neurological conditions that hopefully will be addressed with various cargoes in this capsid.

Speaker Change: All other targets are unclaimed at the moment of course, we assigned people want to take forward Praluent and we have a range of I think there's eight in our current pipeline on the website that we would consider depending on resources taking forward, we're really lucky that.

Speaker Change: The we've been inundated by other companies looking for opportunities to access the capsid.

Speaker Change: And we're being very careful to.

Nathalie Dubois: And then the second question I think you asked was about fabric.

Nathalie Dubois: Natalie, can you update us on where we are with the fabric study please?

Nathalie Dubois: So we have very encouraging data, but if I understand your question, you're wondering if we are still wanting to partner. Yes, so we are very encouraging data and really a remarkable path forward.

Nathalie Dubois: We've agreed with the agency, but it doesn't make sense for us to set up a commercial infrastructure around a single product. So we believe a company with the appropriate commercialization infrastructure would be best placed to take this medicine more quickly and effectively with patients. What's really interesting is the longer we follow the fabric data, the more interesting it becomes. We have patients out at four years; we have several out at two years. We have 17 patients now that have come off of ERT, and none of them show any sign of going back on. And then the more recent data that we got that we describe in this, where we show an improvement in EGFR.

unknown: You know, what's really interesting is the longer we follow the FABRI data, the more interesting it becomes. We have patients out at four years, we have several out at two years, we have 17 patients now that have come off of ERT, and none of them show any sign of going back on.

unknown: And then the more recent data that we got that we describe in this, where we show an improvement in EGFR. So this isn't a biopsy. This isn't a biomarker. This is what's important to patients and what determines the fate of patients, and in some cases, literally their fate. And the EGFR drops year on year in patients with FABRI disease. ERT slows that somewhat, but we've been told no one has ever seen an EGFR that actually statistically improves, both the mean and the median.

Nathalie Dubois: So this isn't a biopsy; this isn't a biomarker. This is what's important to patients and what determines the fate of patients. And in some cases, literally their fate and the EGFR drops year on year in patients with fabric disease. ERT slows that somewhat, but we've been told no one has ever seen an EGFR that actually statistically improves both the main and the median improves. And it really speaks to the clinical utility, as a physician, the clinical utility of this medicine.

Speaker Change: <unk> disease.

Speaker Change: ER key slows that some port, but we've been told no one has ever seen and egfr to actually statistically improves both the mean and the median improves.

unknown: And it really speaks to the clinical utility, as a physician, of this medicine. So we have several companies that we're talking to now. They, too, have got very excited by this EGFR data. We see this as an important medicine. The team's driving goal is to get this to patients. However it takes to do it, we will make sure that this is a medicine because it is making such a difference to patients with FABRI disease.

Speaker Change: It really speaks to the clinical utility as a physician the clinical utility of this medicine. So we have several companies that we're talking to know they too have got very excited by this egfr data.

Nathalie Dubois: So we have several companies that we're talking to now. They too have got very excited by the CGFR data. We see this as an important medicine. The team's driving goal is to get this to patients. However, it takes to do, we will make sure that this is a medicine because it is making such a difference to patients with fabric disease.

Speaker Change: We see this as an important medicine the teams.

Speaker Change: Driving.

Speaker Change: Goal is to get this to patients. However, it takes to do we will make sure that this is a medicine because it is making such a difference to patients with fabry disease.

Operator: Thank you.

Speaker Change: Great. Thank you.

Operator: One moment for our next question.

Speaker Change: One moment our next question.

Yanan Zhu: Our next question comes from the line of Yanan Zhu of Wells Fargo; your line is now open. Oh great, thanks for taking our questions, and congrats on the progress. So maybe a first couple of questions for the Genentech collaboration, specifically on the technology front. I was wondering, are there new engineering features in the zinc-single repressor that Genentech licensed? Is the repressor similar or different with any of the prior or current zinc-single repressor product candidates that have been worked on? And secondarily, I was wondering how deep of a repression can zinc-single repressors achieve, and how much repression is necessary for achieving therapeutic benefit in suppressing power.

Speaker Change: Our next question comes from the line again.

Speaker Change: <unk> Fargo. Your line is now open.

<unk> Fargo: Oh, great. Thanks for taking our questions and congrats on the progress.

Speaker Change: So maybe a first a couple of questions for the Genentech collaboration.

Speaker Change: Specifically on the technology front I was wondering.

Speaker Change: Are there new engineering features in the zinc finger repressor that genentech licensed.

Speaker Change: If the repressor.

Operator: Thank you.

Amy Pooler: Those are all great questions, and Amy, I'm sure you'll love to answer them. Yeah, absolutely. Thank you for the question. The zinc-single repressor technology is something that Sangamo has been working on for some time, and we are really confident in both the potency and the specificity of the platform. This zinc-single, especially for Tao, is really a beautiful example of that zinc-single technology. Now, the thing that was missing in the past for the Tao program really was a way to deliver. We know that these Tao apathys, especially in Alzheimer's disease, affect the whole brain, and with a direct injection approach or an inter-SQL CSF approach, we really weren't able to achieve that widespread brain distribution that we think would be critical for treating the disease.

Amy Pooler: The important thing is to pair that potent and specific Tao zinc-single repressor with the stack-bbb capsid in order to achieve that. As we showed in ASGCT earlier this quarter and also in our corporate deck, we have really outstanding distribution of the stack-BBB capsid throughout key brain regions that are involved in Alzheimer's disease. So, we're really excited about that. So, in terms of that. And I think the other piece that we're delighted about in this is Genentech. Genentech and Casper, the head of neuroscience at Genentech, has always been a group fan of zinc fingers as a way to control gene expression.

Speaker Change: Associates, So we're really excited about that.

Speaker Change: So in terms of I think and then I think the other piece that we are delighted about and this is.

Speaker Change: Genentech Gen Tech and Casper the head of neuroscience of Genentech has always been a great fan of zinc fingers as a way to control gene expression and they have a love and a passion for Alzheimer's we feel in their hands it will get to patients as quickly as possible. So this from a single point of view is.

Amy Pooler: And they have a love and a passion for Alzheimer's. We feel in their hands; it will get to patients as quickly as possible. So, this, from a Sangamal point of view, is getting the right deal with the right partner. And if for us, it validates both our cargo, the zinc finger repressor, and our capsid. And we're delighted to partner with Genentech in this. And then to follow up on the second part of the question about the potency of the repression, we see greater than 90% repression on a cell-by-cell basis in neurons, which are the key cell types that are affected in Alzheimer's disease.

Speaker Change: <unk> is getting the right deal with the right partner and for US It validates both our.

Speaker Change: Cargo of the zinc finger repressor, and our capsid and we're delighted to partner with with Genentech in this.

Speaker Change: And then to follow up on the second part of the question about the potency of the the repression, we see greater than 90% repression I'm on a cell by cell basis, and neurons, which are the key cell types that are affected in Alzheimer's disease. So that demonstrates this potency we see that both in vitro and neurons that we can grow in the dish and also in vivo and in animals.

Amy Pooler: So, that demonstrates this potency. We see that both in vitro in neurons that we can grow in the dish and also in vivo in the animal studies that we've completed. A naming force necessary.

Amy: That is that we've completed and Amy what's necessary.

unknown: Now that's the million dollar question; what we see with the SacPBB repression, mediated repression that we've demonstrated in non-human primates, we believe is at levels that are necessary again on a single cell basis of over 90% in those neurons. So, really excited about being able to move this program into development and get into patients.

Amy Pooler: Now, that's the million-dollar question. What we see with the repression, mediated repression that we've demonstrated in non-human primates, we believe is that levels that is necessary, again, on the single cell basis of over 90% in those neurons. So, really excited about being able to move this program into development and get into patients. That's very, very helpful.

Amy: Now that's the million dollar question I had and what we see with the fact that the repression mediated repression that we've demonstrated in non human primates. We believe is at levels that is necessary again on a single cell basis over 90% and those neurons.

Amy Pooler: If I may ask two additional questions, one on stack BBB, sounds like this capsule really has a lot of very intriguing features, not only for broad neural construction, but also with deep target impressions, deep targeting of deliver. I was wondering, have you undertaken any work to characterize what might be the cell surface target that has been, that is targeted by this engineered capsid?

Nathalie Dubois: And another question on the FABRI program, I was just wondering, hearing about the improvement in EGFR, could you give us a little bit, a little more color on how many patients do you have long term EGFR data for, data for, and also what might be the venue for this upcoming data readout. Thank you.

Nathalie Dubois: Thank you for your questions; great questions again, Amy. Can you talk a little bit stack BBB? Sure, we presented some data on the possible mechanism of blood-brain barrier crossing of stack BBB at the ASGCT Conference earlier this year. What we found was a potential mechanism where it's highly conservative protein in both mouse, non-human primate, and human.

Nathalie Dubois: Of course, the data that we've seen in the non-human primate is really compelling, but we also understand that the most important thing is the proof of principle in humans. And this is really why we're so excited to be driving forward the stack BBB capsid with our pre-on program. And that will give us the potential to make a difference in the license of patients that have this fatal disease.

Nathalie Dubois: Natalie, can you talk about the longer-term data that we have with FABRI please? Yes, so at this point we have 18 patients at one year where we have followed the EGFR, and we can see that there is the mean and both the mean and the median of EGFR is improved when you look at those 18. Statistically significant. It's a significant improvement in those 18 patients. In this patient, we have both male and female. So we're very excited with this data because we demonstrate for the first time an improvement in EGFR, which really does not happen on ERT.

Nathalie Dubois-Stringfellow: Nathalie, can you talk about the longer term data?

Speaker Change: Is improve when you look at those ages statistically significantly.

Speaker Change: Second improving those 18.

Speaker Change: Patient in this patient we have both male and female.

Speaker Change: So we're very excited with this data because we demonstrate for the first time and improvement in Egfr, which really does not happen on the RT and.

Nathalie Dubois: And if you remember, in addition, the patients that are coming off their ERT, their SF36 is positive, they're sweating, just the general symptomatology of these patients is all positive and the capsid is incredibly well tolerated. So we're very pleased, and the patients and the patient's support groups are very pleased with the progress of this answer.

Speaker Change: If you remember in addition, the patients are coming off through your T. There.

Speaker Change: So if there are two six is as positive there sweating. The just the general symptomatology of these patients is is all positive and the capsid is incredibly well tolerated. So we're very pleased on the patients and the patient support groups are very pleased with the progress of this of this.

Speaker Change: So it.

Nathalie Dubois: Thank you. I look forward to seeing the data.

Speaker Change: Thank you looking forward to seeing the data.

Mori Raycroft: One moment for our next question. Our next question comes from the line of Mori Raycroft of Jeffrey. Your line is now open.

Speaker Change: One moment for our next question.

Speaker Change: Our next question comes from the line of Maury Raycroft of Jefferies. Your line is now open.

Mori Raycroft: Hi, thanks for taking my question, and I'll add my congrats on the UNITEC deal as well. I have a question just on a human-feeling A with Pfizer since the $70 million milestone payment is tied to the BLA filing, which seems like a lower risk event for Pfizer. Could you potentially accelerate that milestone payment, and for the commercial milestones, would you consider renegotiating earlier payments for those milestones as well?

Maury Raycroft: Hi, Thanks for taking my question and I'll add my congrats on the Genentech deal as well.

Sandy Macrae: Thanks for the comment, Maury. We look forward to Pfizer moving this forward and to registration and beyond.

Sandy Macrae: We were particularly encouraged by the recent Pfizer, the quarterly coal, where this was a core part of their pipeline and something that they spoke up and were excited about, particularly as it matches with their hemophilia B asset as well. We don't give details of when these milestones will be coming. We don't need to draw down these milestones or royalties.

Sandy Macrae: Of course, when the results were announced, we had inbound inquiries from royalty companies who were interested in talking about that with us, but we're so close to the registration that it's a balance between supporting Pfizer and believing in Pfizer and the need for cash now, and so we are blessed to have that choice. With this money that we've brought in from Genentech, it gives us time and it gives us an opportunity to complete other business development deals. With the February, we have the choice and the results are giving us incoming interest on the clinical benefit with the capsid and cargo that Amy has created.

Sandy Macrae: We can do other capsid and cargo deals.

Sandy Macrae: So this has been a great turnaround from Sangamo.

Sandy Macrae: This is the beginning, and we are fortunate to have so many assets that we can monetize and choose our own path. Got it, it makes us. That's helpful. Maybe one quick follow-up.

unknown: Got it. That makes sense. That's helpful. And maybe one quick follow-up. I'm just wondering if there's more you can say about the stack BBB outlicensing process and the extent of diligence from Genentech and whether other parties were involved in TAO specifically.

Sandy Macrae: I'm just wondering if there's more you can say about the STAC BBB out licensing process and the extent of diligence from Genentech and whether other parties were involved for a toss specifically. There's a limit to what we can say. We saw this data first of its STAC BBB in November in amongst a whole other library. We saw the data on the single capsid in March, and the business development team did a great job of driving it forward. Of course, I wanted it sooner. They will be smiling at me, saying what a great job they have because I was always asking them to do it sooner.

Amy Pooler: Why we particularly like Genentech as our partner is the deep, deep diligence. They've turned over every stone. They work closely with them in her team to make sure that they were making the right choice both with the capsule and cargo. We have several other companies that we're talking to, and we're very careful to make sure that we know what they want as their intended target to make sure we can maximize the value that we can get from this capsid. I mean, it really was significant scientific diligence, wasn't it? Yeah, it was such an exciting process and really such an honor also to go through it with the Genentech team.

Speaker Change: So our partner is the deep deep diligence they've turned over every stone they work closely with Amy and her team to make sure that we're making the right choice both with the capsid and cargo we have several other companies that we're talking to and we're very careful to make sure that they.

Maury Raycroft: We know what they want.

Maury Raycroft: Their intended target.

Amy: To make sure we can maximize the value that we can get from this capsid Amy.

Amy: Really was significant scientific diligence wasn't too yeah.

unknown: Yeah, it was such an exciting process and really such an honor also to go through it with the Genentech team. You know, they're really wonderful looking at the data together and working in such a collaborative way. So, really looking forward to working together.

Amy: Yeah, It was such an exciting process and.

Amy: I'll try to go through it with the Genentech team out there really wonderful looking at the data together and working on such a collaborative by itself really looking forward to working together.

Amy Pooler: They're really wonderful looking at the data together and working in such a collaborative way, so I'm really looking forward to working together.

Operator: Okay, thanks for taking my questions.

Speaker Change: Great. Okay. Thanks for taking my question.

Operator: As a reminder, to ask a question, we'll need to press star 1-1 on your telephone and wait for your name to be announced.

Speaker Change: As a reminder to ask a question you will need to press star one on your telephone and wait for your name to be announced.

Lisa Walter: Our next question comes from the line of Lisa Walter of RBC. Your line is now open. Oh great, thanks for taking our questions. This is Lisa on Feluca. First on February, it has been about 10 months since a search for a partner has started by my count. Sounds like there have been some fruitful discussions going on with strategic and as well as clinical data progress and progress with FDA and EMA regulators too.

Maury Raycroft: Our next question comes from the line of Lisa Walter of RBC. Your line is now open.

Amy: Oh, great. Thanks for taking our questions. This is Lisa on for Luka.

Lisa Walter: But just wondering if you can add any more color on what is holding a potential strategic back here from partnering with you on February. And my second question on Hemophilia A, just wondering how the conversations with Pfizer are evolving on which regions will be most strategic to file a BLA. In the past, they believed the US, EU, and Japan were flagged. However, we saw yesterday that Biomorin is opting to limit their launch now to only three countries: the US, Italy, and Germany. So my question is, will a more focused launch be under consideration? Or do you need to go broad for Hemophilia A considering you'll be a second to market?

Sandy Macrae: Any color here will be helpful.

Sandy Macrae: Thank you.

Sandy Macrae: So I'll answer the second question first. We can't comment and won't comment on Pfizer's marketing plans. That just is not appropriate. And it's also in the contract that Pfizer would be the people who would speak to that. We noted with interest, Biomorin, reducing the number of companies who are intending; you're reducing the investment. It's really hard to relaunch a product. And we're pleased that this gives Pfizer an opportunity to make this medicine available. Our data looks great. The efficacy looks good. The tolerability looks good. And in Pfizer's hands, I'm sure it will be an important medicine.

Sandy Macrae: You asked an interesting question about February. We're determined to do the right deal, not a quick deal; do the right deal. And with the money that we brought in from the Genentech deal, we have the time to work with the right partner to take February forward. What's interesting is, as the discussions have continued, the data has matured. The data has improved. The biopsies are starting to come in in the coming months. The EGFR is statistically significant. And the partners are excited. And we look forward to solving this as soon as possible. It's an important medicine, and it's important that we get it right.

Operator: I am showing no further questions.

Louise Wilkie: I would now like to turn the call back to Louise Wilkie for closing remarks. Thank you once again for joining us today and for all your questions. Ever a reminder, you can access our presentation on the Investor Relations section of the Sangamo website. We look forward to keeping updated on our future developments. Thank you for your participation and today's conference.

Operator: This concludes the program.

Operator: You may now disconnect.