Q2 2023 Sangamo Therapeutics Inc Earnings Call

Okay.

Good day, and thank you for standing by and welcome to the Sangamo second quarter 2023 teleconference call. At this time all participants are in a listen only mode. After the speaker's presentation, there will be a question and answer session.

To ask a question. During this session you will need to press star one one on your telephone you weren't didn't hear an automated message advising your hands. It's right to withdraw your question. Please press star one one again please be advised that today's conference is being recorded I would now like to hand, the conference over to your.

Speaker today, Luis Wilkie, Vice President of Investor Relations and corporate Communications. Please go ahead.

Thank you good afternoon, I'm Louise Wilkie Bank of my Vice President of Investor Relations and corporate communications. Thank you for joining us on the call today.

On this call are several members of the Sangamo executive leadership team.

Sandy Macrae, Chief Executive Officer, Mark Mcclung, Chief Operating Officer, Fritz you should do or Bob Reich, Chief Financial Officer, Jason <unk>, Chief Scientific Officer, not lead you Posturing Feller, Chief Development Officer, and Lisa Rooikat Chief Medical Officer.

Slide small corporate presentation can be found at our website Sangamo dot com under the investors and media section of the events and presentations page.

This call includes forward looking statements regarding <unk> current expectations. These statements include but are not limited to statements related to the therapeutic and commercial potential of our product candidates the anticipated plans and timelines for Sangamo and our collaborators for regulatory submission initiating in conducting clinical trial screening and dosing patients presenting clinical.

Data.

It's about product candidates anticipated feedback from and interactions with regulatory agencies.

A preclinical programs to the clinic, a strategic re prioritization and the anticipated benefits thereof.

Sufficiency of our resources cash runway and plans to seek additional capital estimated financial guidance for 2023 and estimate of 2020 for operating expenses.

Coming catalyst of milestone and other statements that are not historical facts.

Actual results may differ materially from what we discuss today.

These statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC.

On a on our annual report on Form 10-K for the fiscal year ended December 31 2022.

Lamented by our quarterly report on Form 10-Q for the quarter ended June 32023 filed with the SEC.

The forward looking statements stated today are made up of the states.

Take no duty to update such information, except as required by law.

On this call we discuss a non-GAAP operating expenses reconciliation of this measure to our GAAP operating expenses can be found in our press release, which is available on our website.

Now I'd like to turn the call over to our CEO Sandy Macrae.

Luis and good afternoon to everyone joining the call.

The second quarter, it's been a busy one per cent and I'm pleased to share some exciting clinical and preclinical progress as we advance our mission to translate groundbreaking science into genomic medicines that transform the lives of patients afflicted with serious genetic diseases.

Our visionary culture has been a key driver in our success to date alongside our innovative approach to genomic medicines Booth, which I believe will continue to drive single to or forwards towards the future.

Starting with our clinical programs, we continue to make good progress in our phase one two star study for Fabry disease.

Having now dosed a total of 22 patients.

Importantly, we recently received productive written feedback from the ft on our proposed phase III trial strategy.

While we're seeking some specific clarifications, it's as it stands today, we feel we have a regulatory path forward to a potential phase III trial.

I'm enormously proud of the team for the work to get us to this point.

We're refining our trial strategy and expect to submit to a proposed phase III protocol to the FDA as early as the end of 2023.

Needless to say our operations team is driving forward with the logistics of these studies in parallel.

We've also made good progress advancing our wholly owned clinical asset TX 200, our car T. Reg cell therapy candidate for renal transplant rejection.

Based on the encouraging safety data to date, we received endorsement from the safety monitoring committee to progress to the next dose cohort. We are encouraged by the rate of patient movement. We're seeing in this study and are pleased to be receiving initial approvals from European regulators for an updated study protocol that we believe will any.

Plus to accelerate more quickly through the dose escalation phase.

The underlying power of Sangamo is her finger platform, which we believe is at the cutting edge of genomic medicine.

Jason will share more context on the importance of our science and our neurology pipeline shortly along with some of the exciting scientific advancements we presented this quarter on our two lead neurology programs, our AAV capsid onto our platform.

Yeah.

These scientific advancements are at the heart of the three business development deals, we recently signed and Mark will elaborate on shortly.

All of which underscore the important role <unk> plays in helping to progress the broader cell and gene therapy industry forward.

We are proud to be part of the important collaborations as necessary within the field in order to best serve patients and are happy that our genome editing and payload delivery technology is being recognized by the broker industry.

Lastly, I'd like to take a moment to welcome Dr. Lisa ROIC here as our new Chief Medical Officer.

Lisa joins us with strong experiences a position and a drug developer.

I'm delighted to have her as a strategic member of the leadership team focused on advancing our clinical and preclinical programs.

I am confident she will continue to bring great I'll reach of Sangamo as we progress our potential medicines closer to patients.

We are proud of the strong progress we have made this quarter.

Nevertheless, we recognize the importance of bringing in additional funding as well as further reducing our operating expenses.

This is a top priority for me and the rest of the leadership team and remains at the forefront of decision makings, each and every day.

While we stay committed to execution and a prioritized clinical and preclinical activities.

We look forward to providing updates in these areas at the appropriate time.

Okay.

I'd now like to turn the call over to our Chief Development Officer, Natalie who will share more on our clinical programs.

Okay.

Thank you Sandy and good afternoon to everyone on the call. It is my pleasure to provide an update on our clinical program over the last quarter, beginning with fabry disease in May we announced that the U S. FDA granted fast track designation for SG nine 'twenty, our wholly owned gene therapy product candidate for the treatment.

Fabry disease.

Fast track designation is intended to facilitate the development and expedite the review of new therapeutics and reflect the substantial unmet medical need for patients with fabry disease with <unk>.

Leading this important designation reinforces the encouraging data we presented from our phase one two star study to date.

Demonstrate the desire from regulator to provide fabry patients with improved treatment options.

The phase one two star study continued to advance with 13 patients now dose in the expansion cohort for a total of 22 patients dosed overall, we're making good progress in enrollment with strong demand from side internationally.

Anticipate presenting additional phase one two data in early 2024.

This quarter, we submitted the briefing book, describing our proposed phase III trial strategy to the FDA and recently received a written response, something which is increasingly common with the edge.

We were pleased with the clarity of the feedback and feel encourage said we have a regular passport for potential phase III trial and for fabry disease patients.

While we cannot disclose full detail of the strike.

<unk> strategy at this time I can share that we are currently designing a trial that is intended to maximize the potential addressable patient population for SG 920.

Two studies.

Based on the Fda's feedback received we do not expect a head to head comparison with enzyme replacement therapy to be required in the naive and pseudo nice patient studied we're seeking additional information from the FDA on specific point.

And on some specific time expect to submit a protocol phase III protocol.

To the FDA as early as the end of 2020 three.

We hope to be able to provide further clarity on next steps in the coming quarter.

Moving to the phase one two steadfast study of our wholly owned TX 200 car T. Reg cell therapy candidate for the prevention of immune mediated rejection in HLA <unk> mismatch kidney transplantation from a living donor we made some significant progress this quarter.

Total of six study sites across four countries are now open and actively enrolling patients with strong interest being seen across our study sites.

Safety monitoring committee met in May to review data from cohort, one and I'm pleased to share that the endorsed moving to the next higher dose level based on the encouraging safety data from the three dose patient and to control patients.

Preparation for the next dose cohort actively progressed, we have successfully manufactured the product candidate for patients for the first patient in the second cohort and the patient received a kidney transplant dosing is expected in the third quarter of this year.

In parallel we continue to pursue opportunity to accelerate dose exploration with regulators based on the encouraging safety data to date with lately is best for patient to accelerate as safely and quickly as possible through to the highest dose cohort where the dose can be more than 20 times.

Later than the one used in cohort one.

We have received scientific advice and submitted an amendment study protocol to regulatory authority in Europe . We are thrilled to have already received our first full country approval.

We intend to share initial data by the end of the year.

Finally, if you could change could tell Pablo back our investigational gene therapy, we are developing with Pfizer for patients with moderately severe to severe hemophilia. A currently in phase III continues to progress as we outlined last quarter dosing required for primary analysis.

It is incomplete and Pfizer continue to work towards a pivotal readout expected in mid 2020 for Pfizer anticipate potential BLA and MMA suggestion submission in the second half of 2024.

I will now turn the call over to our Chief operating Officer, Mark <unk>, who will discuss business development update from the quarter.

Mark.

Thank you Natalie and good afternoon, everyone.

Core component of the Sagamore business strategy is to drive value by advancing our wholly owned pipeline, while Simon simultaneously strategically leveraging partnerships that create additional value.

We have made a deliberate decision to provide biotech and pharma partners with access to our technology. When they are working in areas outside our strategic focus or when they can bring added value of their own expertise a proprietary technology.

This advances medicines towards patients more quickly.

And this is our priority, but it also accelerates learnings that we can apply to our own programs and technology and brings important non dilutive funding to the company.

The partnership World is opening back up in biotech and as a result, our business development team has seen an uptick in interest in the last few months, we have been excited to announce three strategic partnerships that not only highlight the potential value of our technology, but also the incredible work going on with the related fields.

These transactions reflect continued interest in our technology and importantly, both genome editing and payload delivery are being perceived as something sangamo can offer.

We feel encouraged by this progress and feel honored to be part of such innovation for the betterment of patient lives.

As outlined at the last quarter earnings our neurology platform is at the heart of our prioritized certain strategic focus and we're very proud of the progress that we're making through our own AAV capsid engineering platform called SR.

The advancement of which is essential to solve the current challenges of delivery.

<unk> are critical to unlocking the therapeutic potential of gene therapies, and our entire genomic engineering platform.

Last month, we granted rights to prevail therapeutics, a subsidiary of Eli Lilly to access certain sangamo proprietary cerebral spinal fluid administered capsid for the evaluation of neurologic targets, which we are not planning to study ourselves.

Sangamo earned an upfront payment and if prevail exercises its option for all targets and to prevail product is approved in the U S and Europe for each target Sanger would also become eligible to earn exercise exercise fees as well as developmental and commercial milestones of approximately $1 2 billion. In addition to tiered product royalties.

This agreement highlights the potential value of our AAV capsid engineering program not only for our wholly owned programs, but also as an opportunity to work with partners to broaden the scope of the diseases addressable by AAV gene therapy.

We are thrilled to work with prevail and evaluate our three refineries cerebral spinal fluid administered novel capsid for neurological targets and are pleased that the other genomic medicine companies recognize the potential value.

Each target indication is its own specific brand coverage requirements. So theres no one size fits all cap suite to address all indications.

That's why at the end of June we also shared that we entered into a license agreement with Voyager therapeutics in connection with our epigenetic regulation treatment of prion disease.

Combining voyagers intravenous administered delivery capsid with ours zinc finger transcriptional regulators that we believe can specifically and potently block expression of the prion protein, which is the pathogenic driver of prion disease.

Our goal is to bring this potential treatment to patients with this devastating disease as soon as possible and based on the animal data. We believe voyages capsid is the right one to target the specific parts of the brain that we believe are relevant to prion disease.

This agreement reflects stangl in Voyager shared commitment to addressing unmet need of neurologic diseases and gets us one step closer to our anticipated R&D submission in 2025.

Given the rapidly progressing and fatal nature of prion disease, we invoice or felt it was important to work expeditiously to bring a potential new medicine to patients.

This mutual sense of urgency led to the decision to move forward with voyagers capsid for crayon, while our own significant efforts to discover captures with broad brand delivery.

That will be advanced further sangamo programs.

Finally last month, we announced an agreement with chroma medicine under which chroma will evaluate select zinc finger proteins for epigenetic editing.

Leveraging our technology platform in combination with promus epigenetic regulators demonstrates the power of companies joining forces to advance the development of potential medicines sang.

Sangamo will be providing chroma for evaluation specific zinc finger proteins designed to target the specified collaboration genes, which are outside of the central nervous system and our strategic focus in exchange for upfront technology access payment.

If chroma exercises its options for any or all targets sangamo will become eligible to earn in option exercise payment. In addition to development and commercial milestones and tiered product royalty payments.

We are very happy to explore combining our zinc finger technology with promus capabilities and believe this work will further validate the importance of the zinc fingers as an ideal platform to support epigenetic editing.

The importance of these partnerships stretches beyond potential monetary returns. They are also crucial and critical to unlocking the full value of our scientific assets. We remain committed to our shareholders and we will continue to seek strategic collaborations that enable us to leverage our technologies in order to support our future plans.

I will now turn the call over to our Chief Scientific Officer, Jason for an overview of the preclinical pipeline Jason.

Thank you Mark.

And good afternoon, everyone. It is great to have this opportunity to highlight the work from Sangamo research in our preclinical pipeline in the past quarter.

Today, I will specifically focus on the exciting progress we've made in advancing our neurology focused in vivo genome engineering therapeutics pipeline.

Our strategy to focus on developing transformative medicines for neurological diseases was born out of a decision to maximally leverage.

Core strengths of the Sangamo scientific team and the most differentiated aspects of our science and technology platforms.

Our goal is to develop transformative genomic medicines that are both safe and efficacious.

To achieve this goal our AAV based genomic medicines require two critical factors for success.

A highly precise and potent cargo, namely our zinc finger epigenetic regulators.

And an AAV capsid that can deliver this cargo safely and effectively to the relevant tissues and cells.

Both are crucial for success and we believe Sangamo is uniquely positioned with this combination of cutting edge technologies innovating.

Innovating in these areas is a major focus of Sangamo research engine and as evidenced by the data we presented this quarter at <unk> and elsewhere and by the recent agreements with prevail Therapeutics and chroma medicine, we are making tremendous progress. This progress is bringing us closer to potentially transformative genomic medicines. This.

<unk> is also being recognized by both the scientific field and by others in the biotech and pharmaceutical industry.

The cargo component of our AAV based genomic medicines are from our zinc finger genomic engineering platform.

Sangamo genomic engineering platform has a set of unique properties that in combination allow us to create potential therapeutics.

That would simply not be possible using CRISPR based systems.

<unk> zinc finger transcription or pressors are derived from naturally occurring human genes are compact enough to be delivered to be packaged and delivered and even multiplexed complete with cell type or tissue specific promoters in AAV viral vectors and are able to exert their therapeutic properties without cutting mutating.

Otherwise damaging the genome.

I'm excited by the progress and the new data we are seeing in our preclinical neurology pipeline.

Our recently announced announced NAV one seven program aims to develop a transformative genomic medicine to treat neuropathic pain, using our zinc finger based epigenetic represses targeting the NAV one seven gene.

We are particularly excited about the number of patients that might benefit from this potential medicine theres, an estimated prevalence of at least 43000 patients in the U S for our lead indication for NAV, one seven associated small fiber neuropathy and.

And we believe that there is significant opportunity to expand into other areas.

This quarter, we presented a comprehensive package of data evaluating our zinc finger transcription of Rhopressa is in this program.

This work demonstrated potent and specific repression of NAV, one seven expression at both the RNA and protein level and culminates with data from our neuropathic pain model in mice, demonstrating that in vivo repression of the NAV, one seven gene reverses pain hypersensitivity.

We have also identified human specific lead candidate zinc finger transcription or pressors that demonstrate potent repression of NAV, one seven gene expression and human <unk> derived neurons.

Importantly, we have found essentially no off target activity, including no repression of any of the closely related <unk> channels.

I'd like to highlight this amazing selectivity versus other family members is a major differentiator of our program and why we believe it has the potential to be both efficacious and safe.

Consistent with this our most recent data from nonhuman primate models with human specific lead candidates showed significant repression of NAV, one seven gene expression in the dorsal root ganglion and all analyses continue to support progression to IMD, enabling nonhuman Primate studies, we are on track for unexpected IND submission for.

This program in 2024.

Yeah.

In animal models of prion disease or other lead preclinical program, we've shown that our zinc finger transcription or pressors significantly reduce expression of the prion protein in the brain safely extend the lifespan and limit formation of toxic prion aggregates.

A final selection process for human therapeutic lead candidates is in progress and plans for IND, enabling studies are well underway.

And as Mark mentioned earlier, our recently announced license agreement with Voyager provides a capsid that we believe will help US advance. This important program forward as quickly as possible.

For our NAV one seven in prion programs, we've chosen captains that are available to develop today.

To maximize the delivery of our epigenetic modulators to the appropriate sites in the nervous system for future programs and some of the most valuable neurology indications are new and improved generation of AAV capsid as needed.

Unifying such capsid has been a focus of paramount importance for the entire AAV field in recent years.

Leveraging our long standing commitment and experience in the AAV delivery, we are focused on solving delivery to the central nervous system for a multitude of neurologic targets that are that need both convenient and broad delivery to the brain.

Specifically, we have developed a multiplex transcription dependent directed evolution capsid engineering platform called SR.

Using this unique selection platform, we are seeking to generate an identified novel AAV variance that exhibits significantly improved either widespread or region targeted transduction and expression in the CNS when delivered by either direct injection or through the blood brain barrier when delivered by the blood.

And this last quarter, we presented data describing the identification of multiple novel AAV capsid exhibiting characteristics consistent with enhanced blood brain barrier transit and showing substantial improvement over AED nine when delivered via blood circulation in nonhuman primates.

Also this quarter, we presented new data describing performance optimization experiments that identified second generation variance of our cerebral spinal fluid administered stock 102, capsid that mediate an additional five to 10 fold increase in CNS delivery in nonhuman primates.

I would also like to highlight that based on our real world experience, bringing AAV based therapies from bench to bedside.

We recognize the importance of including considerations related to scale up and manufacturing early and our discovery process in that regard analyses also presented this quarter demonstrated improved manufacturing yields for some of these novel capsid when compared to our benchmarks.

We are evaluating the second generation capsid individually and believe that there are promising candidates for future potential therapies.

As Mark outlined earlier.

There may not be a one size fits all capsid that could address delivery to all potential indications that is why we continue to devote significant resources to developing and characterizing novel AAV capsid variants with enhanced CNS tropism.

We believe that this work will drive significant long term value for sangamo by allowing us to expand the addressable indication space for origin epigenetic engineering technology to include major diseases with high unmet need, including Parkinson's and Alzheimer's disease.

We feel a great responsibility to patients to apply our technology to as many potential medicines as possible.

And this is reflected by the announcements that mark spoke about earlier. The recognition we are receiving from other companies and how we are deploying our zinc finger technology to be combined with the unique capabilities of our partners is a testament to our commitment to our mission from.

<unk> <unk> forward, our own internal programs to enabling our partners. We continue to advance the field of epigenetic editing.

I will now turn the call over to our Chief Financial Officer, Patricia for an overview of the financials producer.

Thank you, Jason and good afternoon.

We ended the quarter with approximately $182 million in cash cash equivalents and marketable securities, which represents a net decline of $59 million from the prior quarter.

We believe our available cash cash equivalents and marketable securities as of June 32023 in combination with other potential cost reductions will be sufficient to fund our planned operations for at least the next 12 months.

We have made some great progress in our business development efforts this quarter as Marc outlined and we continue to proactively explore a range of options to raise additional capital.

non-GAAP operating expenses, which exclude impairment charges and stock based compensation expense for the second quarter ended June 32023, with approximately $72 million compared to 67 million for the same period in 2022.

Primarily due to the restructuring expense related to the reduction post announced this quarter and higher operating costs as we advance our clinical trials.

As a reminder, our current investments have been directed towards our three prioritized areas of focus advancing a fabry program for potential phase III trial.

Progressing TX 200 to two phase two as quickly and safely as possible and the development of a prioritize CNS pipeline.

Our detailed financial results for the quarter are available in the press release and 10-Q issued this afternoon, which can be found on our website.

Turning to our 20, turning to our full year 2020 guidance, we expect our full year non-GAAP operating expenses, which exclude certain noncash impairment and stock based compensation expense to remain unchanged and to be in the range of approximately $2 8 million to $260 million.

As a reminder, the restructuring we announced at Q1 earnings is expected to result in annualized savings of approximately 31 million going forward.

We expect our 2020 for operating expense to reflect these savings and to be significantly lower as we further focus our resources in line with our strategic priorities.

We remain dedicated to advancing that'd be in TX to 100 through the clinic. We're focused on advancing are wholly owned neurology epigenetic regulation pipeline.

I'm very pleased that we've been able to share more of our promising preclinical data in this important area.

Importantly, we continue operate with a strong focus on fiscal discipline and prioritization and recognize the importance of continues to seek ways to bring in additional funding through a variety of potential channels.

Our focus will continue as we reach internal inflection points are in a position to make strategic decisions.

At this time, we would like to open it up for questions.

<unk> please open the lines.

As a reminder to ask a question. Please press star one one on your telephone.

Wait for your name to be announced to withdraw your question. Please press star one one again.

Please stand by while we compiled Q&A roster.

The first question comes from Mare break Croft with Jeffries Your line is open.

I congrats on the progress and thanks for taking my questions.

Uhm four fabry for for some of the fabric feedback can you elaborate on what the two studies in the proposed plan for the phase III would entail in terms of patient populations and are there any other details you can share on site scope and timing of the two studies.

And follow up giving you don't think that had to had verses in New York. He is necessary what would F. D. A uses it as a comparator.

Good afternoon Marie so thank you for your question, so I'm gonna get some help from naturally here, but we're really pleased with interaction we had with the agency <unk>.

Yes, absolutely I think we had a lot of clarity on proposed design and very optimistic about <unk> you know <unk>.

Clinical phase three protocol obtained by the end of the year, So very happy with the progress in terms of your question. We are studying the entirety of the patient population as you know the the patient population in February disease.

Both in symptoms and treatment.

And we are <unk> to address at the entirety. So we had proposed to studies that.

Dress, both the naive and sorta nice population and the patient on the I T.

We are seeking additional clarification on some of the feedback we received just to finalize our our phase three strategy, but we can sure that we do not need to do it yeah T competitor with a nice at a nice patient and.

That we we will <unk> additional detail on the detail of the phase III protocol at a later date.

<unk> I know you know this but the whole process of guessing having these interactions with agency is big you put an adult human they give you their response and you have the opportunity to us for clarification. So it is very.

Very normal for us to go back and make sure we fully understood. What it is we believe they've said so this the response from the agency only came very very recently and therefore, we are still in the middle of that process.

Got it makes sense, except for those two studies what they started at the same time or would be staggered anything additional we can say about about that.

We are doing our very best to get them, both going as quickly as possible and will give you more details on the at the right time.

Okay sounds good thank you very much.

Please stand by for the next question.

The next question comes from Luca <unk> with RBC capital Your line is open.

Oh, great. Thanks, so much for taking my questions to follow up on more assistance. There for February it sounds like you have great alignment with the F D a and I, even sooner naive patients <unk>. However, what's the classical applications.

Oh D. R. T. What is the F D. A asking there again any color will be much appreciated and then maybe only hemophilia a can you just give a sense of how much energy and time is pfizer, putting behind G therapy for hemophilia. It looks to me that they're real excited about there. So Q I'm gonna call anybody any more systematic and maybe a bit less.

<unk> therapy, but will love if you have a different view here and maybe related wondering if you have any plan to monetize that royalty screen extra much.

Thank you that a lot to answer a single question. So I'm gonna suggest we do it in reverse order <unk> could you talk <unk> hemophilia and then can you might be reflect on the the epidemiology of February about the patients around in the patience or no 20 R. T and then Natalie perhaps you can comment on the.

You are two your patience homework, if you start with you ma'am.

That for hemophilia, a pfizer has not changed or guidance. They anticipate having the top line data sometime in the first half of 2024 and they are still guiding on submitting a b L. A in the second half of 2024 the interactions that we.

Have with them are consistent with the fact that they're continuing to move that program for so we're very excited about the continued progress on that.

You know, we're not going to comment right now in terms of any any monetization of the royalties you know we've we've got it on with the royalties and and milestones are in the in the past and if something changes will provide an update but but as we've talked about it as an option for us should we need to extend our cash runway.

<unk> <unk>, yes, yeah. So is it is I think we've talked about before Luka you know if you take a look at the the the database that we had on fabry patients. There about 4000 patients that have got a diagnosis of fabry in this particular database.

What I find striking is that there's at the best case scenario. There's only about 1800 patients that are engaging at any given time with over the course of a year with the with the health care providers and that's measured by at least two to three visits per year.

But at the low end, sometimes it's as low as 1300, so the way I sort of triangulate that is is there's roughly 4000 patients that have had a diagnosis clearly around 2000 have not been prescribed any therapy right now so they're probably just being monitored by the physician.

There was probably about 1800 that have been put an enzyme replacement therapy. Those are the ones that are regularly going in but when you take a look at the compliance rates there were kind of all over the place and so you've got in any given once you've got as low as 1300 patients that are going in on a regular basis and so.

You know when you when you think about the pseudo naive and naive population a substantial number of the patients fall into that particular category. The number of patients that are are compliant with their regular enzyme replacement therapy is actually a fairly small number of the totally diagnosed population.

Ah Natalie how are we going to so you've said that the <unk> will not need a comparator study, which is great news and <unk>. Those are on E. R. T. So <unk> seeking clarification from the F. D. A M. On some specific question in order to finalize not purple sage.

12 protocol for the yeah, Yeah, T patient, but needless to say that we really as in exciting potential passport for both population naive to deny M as well as T. I T patient on the I T and looked at the two patients are are important because we believe that.

Gene therapy will be <unk>.

Much better patient proposition for them a voice I'm huffing to go every two weeks and the studies that we've done the patients that come also B R. T. Their quality of life goes off the field <unk>, they're staying off E. R. T were over here and some of these patients are.

<unk>, so we feel an obligation to address them.

We need to ask some more questions with the agency and we'll let you know as soon as we can and what did you say no that study will look like.

Thanks, so much very helpful.

Please stand by for the next question.

The next question comes from Greg Harrison with Bank of America. Your line is open.

Hey, good afternoon. Thanks for taking the question for the February 8th three trial.

What would you expect a timing can be on the turnaround. After you submit your protocol to F D. A and how does the initial demand for.

Patient might look like.

We have great engagement with the patient support groups the patience and the current clinical trial are coming to us very eagerly to get this medicine. So naturally if we <unk> submit at the end of the year for <unk>.

So the F. D. A we'll have 30 days to review the protocol and then give us some come in or go ahead for the phase three and then needless to say, we are preparing our operation team or having a start as soon as possible. After we have agreement.

C F T on the final protocol.

Gotcha, and then if I could take on more than what what assumptions are baked into your cash runway gardening in the next 12 months and would you consider partner in any of your home.

Program the other supervisor <unk>.

<unk> can you say that one [noise].

Sure.

And a <unk> 182 million.

And we are projecting that it <unk>.

As far as what assumptions and you have to keep in mind that the guidance for the it's for 2023, the 240 to 60 million and the restructuring depleted in M. Q1, <unk>. The executed is expected to this whole thing <unk> savings so.

We haven't shared our guidance get 420, 24, but along with the savings they expect that 10 for 2024 to be significantly lower.

N F for what we're looking at M. S. N you alluded to Nichol right. We're looking at several options are both from them funding perspective, <unk> five G M partnership market opening up.

And also we're looking at how do we further streamline our ethics.

Yeah.

Thanks again for taking the question.

Lee standby for the next question.

The next question comes from Jan along with Barclays. Your line of <unk>.

Thank you for taking my questions just want to clarify the F. T specific attention to <unk> clarification questions from F. T. Eight that's only recording.

T face my childhood is fine.

Alright. Thanks. Thanks for the question is it's we have the opportunity to clarify a pressing the agency tells us and it's it's important to take this opportunity because it's such a an important piece of information. So we were able to go back.

<unk> across the whole range of things to come into town.

Okay is there any like preclinical data or clinical data clarification on quite a request from the F D before the phase three trial.

<unk> I think we can answer that one yeah. So no. There is no preclinical data that needs to be added and we have a clear password on the data for starting the phase three so I think we have clarity on both of those.

Okay, Great and then very quickly regarding to Avi <unk> can you elaborate a little bit like a voyages Ivy administered S. C N S <unk>.

Compare contracts to your in China's C N S pocket a capsule.

So let me let me possible for two Jason, but just before I do that we're very careful to keep the the teams clear that they are working you throw in a <unk> should we are very grateful to <unk> for giving us access to.

<unk> have to be very careful to note uhm.

Completely information from them together, so very important partnership point to make Jason.

Yeah, so regarding the Voyager caps it in that partnership to develop a therapy for prion disease, you know we've been <unk>.

Extremely excited about the the data that we've generated through our collaboration with the broad Institute and M. I T. In animal models of prion disease, and and we've received really uniformly positive feedback from the community on on the potential there.

And when we saw the the progress that Voyager made in their Ivy administered capsid. We believe that it was really the best way to get that drug to patients and the quickest way possible and if you understand the need in in prion disease I'm sure. You'll appreciate why we felt it was so important and this is a.

Devastating disease, where where people can die extraordinarily quickly and so our motivation there was really around getting that to patients as quickly as possible and and we think Voyager for their for their partnership there is they also recognize that.

Nonetheless, we also have a a major effort in developing our own <unk> and we've seen a lot of progress there I mentioned the the work that it we've we've shown at at a S. G. C. T. We continue to make lots of improvements and we're very optimistic that over the <unk>.

Course of the next year or two we're gonna see some some major developments from our own programs and so as we look at our pipeline we reflect on when we need to commit to a cap said, what <unk> are available and we make choices and a in a strategic manner too advanced the pipeline recognizing that we have to <unk>.

Combine <unk> with a cargo and in each case could be unique and we need to maximize our probability of success, but also maximize our potential to move things forward in a reasonable amount of time. So it's a it's a more multifactorial decision, but you know we we believe that we've got our own <unk> I think that the.

The the partnership with prevail and Lily emphasizes that others in the community are recognizing the strength of our own caps of development program, but at the same time, if there's a need to move something forward more quickly we're always open to doing that.

Thank you very much.

Standby for the next question.

Our next question comes from an <unk> with <unk>.

<unk> Your line is open.

Hi, there thanks for taking our questions firstly on the <unk> program, how little patience are you planning to do as in the phase one two expansion call Hot.

And does he have do I want to see any additional data from these patients before possibly finding authenticate three two the two faced.

<unk> designs and I have a follow up.

Natalie can you do with them.

Yes, so we are continuing to enroll patient and dose patient in the face when to study, but we we do not depend on this data to start up phase three plan and protocol.

Is there any additional data that we need for.

<unk> three trials is something we are not Chang at this point that will provide clarification at a later point I think it's important to realize that this could be a phase one two studies of up to 32 patients.

And it will be incredibly supportive when we find <unk> from the fees three data because those patients will be.

I am showing benefit we we believe for years and that will give that uhm <unk>. The agents <unk> very pleased to see.

Yeah, they will be part of the durability and safety database for daily filing.

And then secondly on T. S 200 could you give us some center for text based on the initial data data for you and will there be any biopsy data and if so what what are you looking for thank you.

Lisa can you to this one.

Sure. Thanks for the question. So as it is a primarily a safety and Tolerability study, we would be you know presenting some of that data at the end of the year and we're not going into details about what additional data we may may present as well.

Please stand by for the next question.

The next question comes from Nicole <unk> with true with your line is open.

Hi, good afternoon. Thanks for taking my question and congrats ninth process.

Two quick ones on that credit your request form so a couple of investors.

Especially considering that the immunosuppressive agents given to patients may surprise style to your production can you help us understand the use of an immuno suppressant Felicity after checks 200 infusion.

And my second question is yeah, there's a lot of interest in the I P. D space from Big for a man and several companies with Cacciari telephones or primary off their I D. D program to Big Pharma can you just remind us the progress with your car to your rank program and diabetes and potential timing for Andy.

So I'm Gonna suggest Lisa takes the clinical technical question Mark do you want to talk about.

<unk>.

The interest there isn't to Redfin Journal believes.

Police police it goes first.

Yeah sure. Thanks for the question so your.

We are using standard immuno suppressive therapy Regiment, and the study and Uhm. Following the you know adaptation in those regiments overtime, so and we haven't actually heard that there have been any concerns related to the the regimen that we're using cause it's it's standard of care extend it.

Practice sewer following you know in terms of the the clinical outcomes looking at evidence of rejection as well as the patterns of immunosuppressive therapy, you by the investigators.

Alright, so in terms of in terms of interested in T. Right cause we've had a lot of inbound interest from potential partners for the T rugs, and I think because we've communicated before.

We have a lot of investors that are looking for opportunities to create a mechanism for them to invest directly into the car T right platform because of that potential and obviously our leadership in that and so we are we are looking at both of those were having conversations with partners, but we're also taking a look at mechanisms to allow for.

For direct investment and will provide inappropriate update at that time, but but yes. There is a lot of interest in the car T rags and the progress that we're making and at this point, we still feel that were the furthest along in terms of the dosing of patience and and Uhm is Natalie commented on <unk> to provide an update in terms of what data we're actually.

Visa said, what what what data will essentially be able to show by the end of the year and that's what we hear from the from the people to come and talk to us about it because we are the furthest along with the only one that talks about being in the clinic. We are the only one with G. M. P manufacturing info bone that allows us to control the process.

We are the only one with the nitrogen capability and we are the one that's got the experience of doing <unk>, that's why <unk>.

They like what they're seeing a sign Google and the T. Rex and we do look forward to sharing the data <unk>. So this is a safety study first and foremost. This is the first time car T. Rex are being given and is remarkably well tolerated.

Great. Thank you so much.

Please stand by for the next question.

The next question comes from Union Zoo with Wells Fargo Securities. Your line is open.

Alright, thanks for taking the price of the <unk> <unk> so two per cent.

First of all you've done so for the space I want to start two O E. R. T re throat patience Freeman F E. R. T and then the second one is 162 hundred so.

50.

What does the company needed to see to move the program for work and how many patients of data would be required for the company to make sure.

Thank you.

Thank you it's always for your question naturally February uneasy one.

So yes, all all the patient in on the I T and the face went to that at least one E. I T remain of the I T. So I I think that was the first question and the second question was.

Okay.

Yeah. So your question was about what safety data do we need to see to move the program forward well, thus far as Natalie mentioned, we've already dose the first three patience and the first dose level and there were no D. L. Ts and we were how to clean opinion from the S. M C to open the second dose.

<unk> and now we're expecting to dose that patience within this quarter I'm. So sorry within the next quarter and I think that I can say that we've been very encouraged by the safety data that we've seen thus far and this is also uhm allowed us to move forward with the protocol amendment to.

Potentially advanced patients more quickly through the different dose levels. So these are all encouraging in terms of longterm, we'll we'll have to see how things evolve on the study.

Got it thank you so much.

Please stand by for the next question.

The next question comes from Patrick <unk>.

True too with the H C. Wainwright your line is open.

Good afternoon, I'm just a few follow up questions for me. The first one is just that you can discuss what remains to be completed and then at 1.7 program, particularly on the manufacturing side of the application ahead of an anticipated.

The submission in 2024.

And then secondly, just you know comments earlier in the call regarding increased interest from potential collaboration partners. I'm wondering if you can discuss what aspects of the platform you receiving the most interest on in what form.

Potential future collaborations could take and if there are specific programs and earlier stage development that you might look to partner.

So not quite can you talk about what's left to do enough 1.7, and then J symbol.

We'll talk about his passion for the platform. Yeah. So we're really excited about the 9117 and really getting to our I D. 2024. The two things that are the two main thing that remains to be completed is the G. L. P talks to be able in a nonhuman primate and this is well.

Planning underway to be completed for 90 next year.

And in terms of the manufacturing component and also we are underway to manufacture the clinical lot to be on time for the first patient in the trial. So at this point the team is working <unk> to to stick to the timeline.

But we foresee no delays and naturally the although we still have to do the G. O P talks we've already done the <unk>, yes, we have done those matching study. So this is you know the normal development password, we need to have the <unk> and the <unk>. There was no absolutely it was beautiful okay.

<unk> Netflix.

Jason.

Yeah. So in terms of an inbound partner interest it covers a wide area, we've already talked about our T. Red platform, we've seen lots of interest from from many different potential partners in in our advances there and particularly around.

You know the capabilities that we built there that go all the way from you know designing cars to manufacturing cells, and then running running the trials in patients. So lots of interest around T. Regs. There are a variety of indications there some of them very large and we recognize that we can't.

Run all of those studies ourselves. So we're we're always in discussions on that front.

I'd say the next the next area, where there's been quite a lot of discussion is around our our zinc finger platform. One of the the interesting things about S. G. C. T. This year was that there seem to be kind of a.

<unk> Ah renewed discovery of something that I think we had sangamo kind of have always known as true and that is the value of the zinc finger platform for developing therapeutics based on you know the real tune ability the specificity the the compact nature of the proteins and so we've really had.

A renewed interest from lots of different companies with different platforms that want to Ah pear their technologies with our zinc finger programs one way that we we kind of evaluate those programs are are those collaborations is how they fit into our strategy. So.

We've talked about a lot today moving forward, we have a strong focus on in vivo genome engineering in the neurology space. So you know one of the first places that were always very open to platform are always very open to partner is to enable companies that are working outside of the.

Neurology space and providing them with the cargo are using finger cargo for for their programs.

When things get into the neurology space, we have to think through in a in a in a smart way what what areas do we really want to focus on where can we execute the soonest to drive value for the company and if there are areas, where we feel that there are other companies that can move things forward quickly and that would ultimately benefit both patients and sangamo.

And we're always open to those types of collaborations.

And then I would say the last area I'm also that has been highlighted by the the deal with prevail is the work that we've been doing on our caps that evolution platform. This is an area that over maybe the last two years, we've been seeing lots of really great data from the team.

And things are progressing very fast and we're very excited about it and it's an area of that as we mentioned earlier the whole field is looking for these types of <unk> because there are lots of diseases that need the right delivery platform and if there's a if there's an indication that we're not going to pursue ourselves, but someone else is interested in.

Doing it and we have a capsule that's able to facilitate that and we're happy to have those discussions. So really you know I'm tremendously proud of all of the work that's going on in the research in preclinical teams to Blue <unk>.

[noise] finger platform forward are captured evolution platform forward. The T rag platform lots of advances and I think we're we're getting inbound interest in all those areas. Most said, Jason and I know you spent a lot of your time talking to prospective partners and.

Are always talking about how excited they are by the sign C C.

Alright, thank you so much.

I show no further questions at this time I would now like to turn to call back to Luis will keep for closing remarks.

Thank you once again for joining us today and for your questions. As a reminder, you can access the earnings release and presentation on Investor Relations section of the <unk> on our website, we look forward to keeping you updated on a future developments thinking.

This concludes today's conference call. Thank you for participating you may now disconnect.

Mmm.

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