Cartesian Therapeutics Inc (RNAC) 2021 Q3 法說會逐字稿

Good morning, and welcome to the Selecta Biosciences Third Quarter 2021 Financial Results and Corporate Update Conference Call. (Operator Instructions) This call is being webcast live on Investors and Media section of Selecta's website at www.selectabio.com, and it is being recorded.

For opening remarks, I would like to introduce Kevin Tan, Chief Financial Officer of Selecta. Please go ahead.

Thank you, and good morning. Welcome to our second quarter 2021 financial results and corporate update conference call. The press release reporting our financial results is available in the Investors and Media section of Selecta's website, www.selectabio.com, and the quarterly report on Form 10-Q ended September 30, 2021, which was filed today with the Securities and Exchange Commission, or SEC. Joining me today are Carsten Brunn, President and Chief Executive Officer; Peter Traber, Chief Medical Officer; and Kei Kishimoto, Chief Scientific Officer.

During today's call, we will be making certain forward-looking statements, including, without limitation, statements about the potential safety, efficacy and regulatory and clinical progress of our product candidates; financial projections; and our future expectations, plans, partnerships and prospects. These statements are subject to various risks that are described in the filings made with the SEC, including the most recent quarterly report on Form 10-Q. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of today, November 9, 2021, and Selecta disclaims any obligation to update such statements even if management's views change.

I would now like to turn the call over to Carsten Brunn. Carsten?

Thank you, Kevin. Good morning. I appreciate everyone joining us today. In the last quarter, we made significant advancements that propelled our business and our existing pipeline forward, which has the potential to overcome immunogenicity, mitigated unwanted immune responses against enzyme or gene therapies, and restore cell tolerance in patients with autoimmune diseases.

We're encouraged by the numerous strategic collaborations that have further validated our ImmTOR platform, designed to mitigate unwanted immune responses across a range of diseases, and by the opportunities to advance next-generation therapeutics. Further, these partnerships demonstrate the broad application of ImmTOR, and we look forward to maximizing the value of our platform as we build on this momentum.

This is a truly exciting time for Selecta. And yesterday, we announced top line results from the first-in-human Phase I dose escalation trial of SEL-399, which we conducted jointly with AskBio. SEL-399 is an AAV8 empty capsid, or EMC 101, containing no DNA combined with ImmTOR at doses of 0.15 mg per kg and 0.3 mg per kg. The randomized, placebo-controlled and double-blind dose-escalation study conducted in healthy volunteers was designed to determine the dose regimen of ImmTOR to mitigate the formation of neutralizing antibodies, or NAbs, against an AAV8 capsid used in gene therapies.

A total of 23 healthy volunteers were enrolled: 14 males and 9 females. All subjects had an anti-AAV8 NAbs titer of less than 1 to 5 at baseline. The primary endpoints evaluated were safety and NAb -- and anti-AAV8 antibody titers. We observed a strong immune response in humans administered AAV8 empty capsids. The peak median anti-AAV8 Nab titer was 1:6,875 at 14 days after infusion, which was maintained at high levels through day 90.

The addition of ImmTOR to administration of empty capsid was observed to reduce the formation of anti-AAV8 NAbs in a dose-dependent manner at day 30. Median day 30 titers of anti-AAV8 NAbs were a 50-fold and 250-fold lower in the 0.15 mg/kg and 0.3 mg/kg ImmTOR cohorts, respectively, compared to the median titer of controlled subjects dosed with AAV8 empty capsids alone.

At 30 days, subjects who received 0.3 mg per kg of ImmTOR, 6 of 6 or 100% exhibited an anti-AAV8 NAb titer of 1 to 25 or less, and 4 of 6 or 67% had a titer 1 to 5 or less. At 30 days, subjects that received 0.15 mg per kg of ImmTOR, 6 of 9 or 67% exhibited an anti-AAV8 NAb titer of 125 or less, and 2 of 9 or 22% had a titer of 1 to 5 or less. At 90 days, 2 of 6 subjects in the 0.3 mg per kg cohort were observed to have sustained control of NAbs with titers of 1 to 25 or less.

Consistent with preclinical data, we observed that the single dose ImmTOR cohorts saw delayed formation of NAbs, eventually reaching similar median levels of NAbs to the control group by day 90. Based on prior animal studies, we believe that if NAbs are inhibited at day 30, administration of 2 additional monthly dose of ImmTOR has the potential to maintain control of NAbs beyond day 90.

From a safety perspective, there were no unexpected AEs related to ImmTOR. The most common AE was mild to moderate dermatitis, which was ameliorated with steroid mouthwash treatment. We believe this data demonstrate ImmTOR's potential to address one of the biggest current mutations in gene therapy, the inability to re-dose life-saving gene therapies due to the formation of NAbs against AAV capsids.

We look forward to leveraging these findings across our wholly owned gene therapy pipeline and alongside our world-class gene therapy partners to achieve our goal of improving the lives of those living with monogenic disease. The press release and webcast with additional detail can be assessed -- accessed in the Investors and Media section of our website.

Next, I would like to highlight our recent business development activity and provide an overview of the key collaborations that have expanded our pipeline of novel therapeutics in combination with our ImmTOR platform. We entered a strategic licensing agreement with Takeda, a global pharmaceutical leader with expertise in rare diseases, to develop targeted, next-generation gene therapies for 2 indications within the field of lysosomal storage disorders.

Together, we look forward to overcoming barriers to current efforts in AAV-driven gene therapy as well as striving to address immunogenicity constraints to enable re-dosing of potentially life-saving gene therapies. Under the terms of the agreement, Selecta is entitled to receive an undisclosed upfront payment and up to $1.124 billion in future additional payments over the course of the partnership that are contingent on the achievement of development or commercial milestones. Selecta is also eligible for tiered royalties on future commercial sales.

We are encouraged by the strong endorsement from large pharma and from other major players in the gene therapy space. To further address key hurdles in gene therapy, we announced an exclusive strategic license agreement with Genovis, to advance IdeXork, or Xork, a next-generation IgG protease to enable the dosing of transformative gene therapies in patients with pre-existing AAV immunity and treat certain IgG-mediated autoimmune diseases.

While most IgG proteases are derived from human pathogens and have a high prevalence of pre-existing antibodies, Xork is derived from streptococcal bacterial strain that does not infect humans. The combination of Xork and ImmTOR has the potential to both mitigate pre-existing antibodies to AAV, expanding access to gene therapy to a vital range of patients and prevents de novo immunogenicity, keeping patients eligible for re-treatment.

Additionally, bacterial-derived IgG proteases are themselves immunogenic. Currently, IgG proteases can only be administered once due to the formation of high-titer antibodies against the protease itself. The combination of Xork and ImmTOR is further differentiated by the potential of ImmTOR to mitigate the immunogenicity of Xork and enable re-dosing of the enzyme, an important benefit for the application of IgG proteases in autoimmune diseases mediated by pathogenic autoantibodies.

In relation to our enzyme therapy program, we announced a partnership with Ginkgo Bioworks, or Ginkgo, to design novel enzymes with transformative therapeutic potential to advance treatments for orphan and rare diseases. This partnership is built on our COMPARE trial of SEL-212 in chronic refractory gout, which provides proof-of-concept data related to into ImmTOR's effect when combined with immunogenic enzymatic therapies.

Under this agreement with Ginkgo, Selecta gains rights to develop and commercialize select therapeutic enzymes from their advanced organism engineering platform to treat autoimmune diseases. We expect that our ImmTOR technology, in combination with Ginkgo's high-throughput enzyme discovery, design and screening capabilities, will bring us one step closer to improving the sustained efficacy of novel biologic therapeutics.

Finally, we established a protein engineering collaboration with Cyrus to radically redesign protein therapeutics. The lead program in the collaboration is a proprietary Interleukin-2, or IL-2, protein agonist designed to selectively promote expansion of regulatory T cells, or Tregs, for the treatment of patients with autoimmune diseases and other deleterious immune conditions. Preclinical data investigating the effect of ImmTOR, in combination with an IL-2 mutein, demonstrates substantial synergistic activity in increasing the percentage and durability of Treg expansion in the spleen.

This supports the potential of ImmTOR, in combination with IL2 proteins, to restore immune tolerance to other antigens and forms the basis for this partnership. Collectively, these strategic partnerships provide additional validation and further demonstrate the robust value of our ImmTOR platform. This is truly an exciting time for Selecta. And with our unique approach and strong cash position, we're well-equipped to maintain our leadership position in the targeted immune tolerance fields.

I'll now take us through some of our key pipeline updates and upcoming milestones. First, a few key points on our enzyme therapy program, which has established an important framework for our clinical development path. SEL-212 was licensed to Sobi and is comprised of ImmTOR, co-administered with our proprietary uricase, pegadricase, for the treatment of chronic refractory gout.

As a reminder, our Phase III DISSOLVE clinical program kicked off in the third quarter of 2020 and consists of 2 double-blind, placebo-controlled trials of SEL-212. In both trials, SEL-212 will be evaluated at 2 doses of ImmTOR on 0.1 milligrams per kilogram and 0.15 milligrams per kilogram; and 1 dose of pegadricase, 0.2 milligrams per kilogram. Each trial aims to enroll 105 patients, with 35 at each dose and 35 on placebo. Enrollment is progressing on schedule, and top line data from this trial is expected in the second half of 2022.

We intend to leverage the success of SEL-212 in for a second enzyme program indication in IgA nephropathy, or IgAN, which is a kidney disease that occurs when immune complexes of an antibody called immunoglobulin A1, or IgA1, accumulate in the kidneys. Current treatments fail to address the root cause of the disease, and we believe our novel approach, which combines ImmTOR with IgA1 protease, has the potential to remove injured IgA from the kidneys and improve markers of renal dysfunction. We remain encouraged by the strong preclinical data in IgA and expect to file an IND for IgAN in 2022.

Returning to our gene therapy program. As noted, immune responses to AAV gene therapy have historically precluded the ability to safely re-dose AAV gene therapies and are a major consideration related to safety and efficacy in the space. The ability to inhibit the development of AAV-specific antibodies has the potential to be transformational and shift the treatment landscape.

We continue to advance our proprietary gene therapy program and filed an IND for our lead gene therapy candidate, SEL-302, which is a combination of MMA-101 plus ImmTOR. MMA-101 is an AAV gene therapy vector for the treatment of methylmalonic acidemia, or MMA, a rare metabolic disease in which the body break down certain proteins and fats.

As of the filing of this Q, the FDA's 30-day review period for R&D to conduct a Phase I/II clinical trial of our SEL-302 product candidate in pediatric patients with metabolic acidemia has expired. However, we have been informed orally by FDA that they're still considering certain aspects of our filing related to chemistry, manufacturing and control, or CMC. We intend to wait for formal clearance from FDA before initiating the proposed Phase I/II clinical trial.

Our second wholly owned proprietary gene therapy candidate, SEL-313, is being developed to treat ornithine transcarbamylase, or OTC, deficiency. OTC deficiency is an x-linked genetic disorder caused by genetic mutations in the OTC gene, which is critical for proper function of the urea cycle. We expect to file a clinical trial application, or CTA, and/or an IND in 2022. We submitted a pediatric investigation plan, or PIP, for SEL-313 to the European Medicines Agency Pediatrics Committee in February 2021.

Overall, we're seeing excellent progress across our gene therapy program, and we look forward to providing further updates later this year as we continue to address the immunogenicity constraints in AAV-driven gene therapy as we strive to overcome repeat dosing limitations by preventing the formation of NAbs, and as we enable more doable and robust expression of the transgene after the first dose.

Now moving on to our autoimmune program. Our autoimmune program is advancing through IND-enabling studies, and we expect to file an IND in primary biliary cholangitis, or PBC, in the second half of 2022. PBC is a chronic, progressive autoimmune liver disorder that leads to inflammation, damage and scarring of the small bile ducts.

PBC has a well-defined target antigen, significant unmet medical need and is well-suited to the application of our ImmTOR platform. Autoimmune disease affects more than 24 million people in the U.S. alone, and we look forward to expanding our pipeline as we continue to explore additional applications of our ImmTOR platform.

Before we turn to the financial results, we have one final update to share. As we execute on our clinical and corporate initiatives, we continue to add talents and depth to our leadership team, and are excited to welcome Kevin Tan as Chief Financial Officer. Kevin brings deep financial expertise and experience in the gene therapy and rare disease landscape.

His impressive track record in capital management and financings, in both the biotech and investment sector, will be invaluable as Selecta continues to pursue new partnership opportunities and advance multiple assets to the clinic. As mentioned earlier, we're extremely excited about the continued growth of our company, and we remain confident in our platform.

Now I'll turn the call over to Kevin to run through our financial results for the third quarter ended September 30, 2021.

Thank you, Carsten. We remain well-capitalized, with $140 million in liquidity as of September 30, 2021, which compares to $140.1 million in liquidity as of December 31, 2020. We believe our liquidity will be sufficient to meet our operating requirements into the second quarter of 2023.

Net cash used in operating activities was $28.9 million for the 9 months ended September 30, 2021, as compared to $42.1 million of cash provided by operating activities for the same period in 2020. Revenue recognized for the third quarter of 2021 was $24.4 million compared to $4.6 million for the same period in 2020.

Revenue of $24.3 million was recognized under the license agreement with Sobi, resulting from the shipment of clinical supply and the reimbursement of costs incurred for the Phase III DISSOLVE clinical program. The significant revenue increase is the result of the continued enrollment of the Phase III DISSOLVE clinical program that was initiated in the third quarter of 2020.

Additionally, during the third quarter, Selecta recognized $0.2 million for the shipment of -- under the license agreement with Sarepta. Research and development expenses for the third quarter of 2021 were $21 million, which compares with $14 million for the same period in 2020. During the quarter ended September 30, 2021, there was an increase in expenses incurred for preclinical programs, salaries, headcount and AskBio collaboration costs.

General and administrative expenses for the third quarter of 2021 were $5.4 million, which compares with $4.4 million for the same period in 2020. The increase in costs was primarily the result of salaries, professional fees and stock compensation expenses. For the third quarter 2021, we reported a net loss of $17.9 million or a net loss per share of $0.16 compared to a net loss of $9.7 million or a net loss of $0.09 per share for the same period in 2020.

I will now turn the call back to Carsten for closing remarks.

Thank you, Kevin. In summary, the promising results from the pioneering Phase I SEL-399 clinical trial suggests that ImmTOR, in combination with a widely used AAV8 capsid, has the potential to overcome the significant challenge of the formation of anti-AAV8 NAbs. Reduction of capsid immunity could be transformational for the field of gene therapy by making gene therapies safer and possibly enabling repeat dosing.

Reflecting on this past quarter, we have made significant steps to progress our existing pipeline candidates and establish collaborations to maximize the potential of our ImmTOR platform. We look forward to providing additional updates on near-term catalysts as we continue to drive our business forward, unlock the power of biologics and address unmet patient needs. I would also like to thank the entire Selecta team, our investors and the many people who have been supportive along the way, including our patients and their families.

With that, we're happy to take questions.

(Operator Instructions) Our first question will come from Kristen Kluska with Cantor Fitzgerald.

The first one is that yesterday's data answered a very important question related to your platform potential in gene therapies. So big picture, looking ahead into next year, what questions or areas in the pipeline do you expect to receive even more clarity on the potential of ImmTOR, whether it's related to your preclinical programs as well as some of the clinical work that's being conducted by both yourself and partners?

That's a great question, Kristen. Obviously, we continue to advance our gene therapy programs. So we hope to be in the clinic with our MMA program next year and, at least, have some preliminary data around biomarkers of the disease and antibody titers. I think that's going to be a key readout.

We have a major readout for enzyme lead program in SEL-212, where we expect Phase III results in the second half of next year. And obviously, we're moving forward a number of the programs that are currently in the pipeline, such as, for example, the OTC deficiency program. We already filed -- plan to file an IND next year.

But we're also moving forward some of the exciting programs that we just struck partnerships with, specifically Xork and IgG protease, which addresses another large unmet medical need in gene therapy. As you know, Kristen, up to 30% of patients have been exposed to a natural AAV infection and have pre-existing antibodies are not eligible for treatment. So that's -- we're going to move that forward next year as well. So next year will be an exciting year for us.

Okay. And now that you have clinical evidence for both the enzymes and gene therapies, and yesterday, you made it a point that the extensive preclinical work really helps with the design and understanding these results. So looking ahead into autoimmune now, as those candidates near going into the clinic, how have you thought about these synergies from a preclinical standpoint in order to best design a future clinical study with the greatest probability of success?

Yes, that's a great question. And obviously, with now over 300 patients dosed with ImmTOR, we have very good safety database, which is always very important, that, we've seen that yesterday, translates very nicely to gene therapy. And I think it will be extremely helpful as well for autoimmune programs as well. We'll provide more guidance early next year.

But I can tell you, we're extremely excited about our partnership with Cyrus, where we released some initial data a couple of weeks ago, where we saw very strong synergies between an IL-2 mutein and ImmTOR, where we saw a higher percentage of overall Tregs when combining those 2 approaches. I will provide more data next year. And I think that would be really transformational for the autoimmune field by really enhancing the tolerogenic effects of ImmTOR.

Our next question will come from Raju Prasad with William Blair.

I was hoping you could provide a little bit of clarity on the assay used to measure neutralizing antibodies here, and when we might anticipate seeing kind of total antibody concentration and IgG levels from this study -- from the 399 study?

Yes. Thanks, Raj. I'll let Kei, our Chief Science Officer, address that question. Kei?

Yes, the assay to measure neutralizing antibody is a cell-based assay. So it's pretty standard within the field. Basically, you're measuring the activity of sera samples to neutralize AAV from transducing hepatocyte cell line in vitro. With respect to the other data, we haven't guided on that. But we are planning to release additional data from this study in conjunction with our partner, AskBio, at an upcoming gene therapy meeting.

Great. And then the dose here was in the 10^12 range. Can you maybe just comment a little bit on how you would think about doses in like the 10^13 or even 1x10^14 range? Do you think that the 0.3 dose might be necessary in some larger capsid doses? Or do you think that data at 0.15 with multiple doses could potentially work for any therapeutic range of AAV that's being kind of utilized right now in the clinical trials?

Yes. So we have -- we've shown yesterday, we have data in mice where we use doses of 2E13 of the AAV vector. Our partner, Sarepta, has dosed even higher. Unfortunately, we can't disclose their data. But in the mouse study, we saw good results as well. We haven't guided which dose we plan to take into the clinic. But I think yesterday's data is a good indication we saw, which was very important to us, a good dose response curve starting at 0.15 mg per kg and then very strong efficacy at 0.3.

But also, I want to remind you that we actually have even tested a higher dose of ImmTOR in the Phase I/II study of SEL-212 as well. So there is still some dose elasticity if we really wanted to push the dose. But I think the 0.15 is a good starting point in our view. And definitely, the learning from the nonhuman primate studies and yesterday empty capsid study is that 3 monthly doses is likely the way to move into the clinic.

Great. And maybe just one last one. I think we've got some questions on kind of the rebound in neutralizing antibodies post day 30. Can you maybe just address a little bit of that? How -- from preclinical studies, how you know that 3 doses will be enough? Is it on like capsid half-life or something like that to kind of enable re-dosing and kind of tamp down antibody levels over the long term?

Yes, that's an excellent question. And obviously, we saw yesterday very good comparability between the data from the nonhuman primate study and the first human study, empty capsid study. Specifically, when we look at day 30, the distribution, the data pattern was very, very similar. And we know from the nonhuman primate study that 3 monthly doses maintain control out to day 90.

We know from the mouse studies, we can maintain their control out to day 168, which is obviously very encouraging as well. So I think we have a good evidence that 3 monthly doses maintain control beyond day 90. We also have -- Kei has done some work with outside collaborators that demonstrated that ImmTOR actually extends the half-life of the capsid. But we believe, past day 90, there's no more capsid, no more antigen presence, actually.

Our next question will come from Gil Blum with Needham & Company.

So it looks like you guys have a -- quite a lot of strategic collaborations. Is there any particular guidelines as to who you partner with, particularly for your gene therapy side of programs?

Yes, Gil, that's a great question. Yes, we've been busy. We've struck 4 partnerships, but we've been highly selective. As you know, we struck a partnership last year with Sarepta as we see them as a leader in neuromuscular disorders. And we're making good progress in that partnership, which is, last quarter, received a $3 million milestone payment. So we're pleased with that.

We're also excited to be working with Takeda. We're working specifically, we're exclusive licensing ImmTOR to -- for 2 lysosomal storage disorders. So these are liver-based diseases. And Takeda is clearly a leader in the space, having a couple of ERTs and some replacement therapies and really investing in their gene therapy business.

So we're very excited. They're committed. They are a long-term player. That's the reason we partner with them. So that's, if you like, a selective out-license deal of ImmTOR. But we've also in-licensed in the gene therapy space, an IgG protease from Genovis, which, we think, is highly differentiated.

And we really want to use Xork to address patients with pre-existing immunity, which really expands the patient pool eligible to gene therapy. So you can see, we've been fairly selective. And we're trying to strike a balance here between selectively license ImmTOR indications that we don't want to pursue ourselves, but also in-licensing assets in -- such the IgG protease from Genovis.

Maybe a related question is [higher]. How do you prioritize your own internal pipeline?

That's a great question. Obviously, we're in a fairly strong financial position, and we really want to have as many shots on goal. If you look at our pipeline, we have, all the way from late-stage Phase III to -- about to enter the clinic to more discovery stage. So obviously, there's different costs associated with that.

And so I think, right now, we do have the means and the resources to move all those programs forward. But obviously, some are more cost-intensive and more resource intense once they move into the clinic. But our goal is to take advantage of the cash position and have as many shots on goal as possible.

All right. And maybe a last one on your IgG and -- I mean (inaudible) is like a compound. You mentioned that there's an issue with re-dosing of these kind of enzymes. And I know that -- I think (inaudible) is a commercial product. How much of an issue is this?

Yes. So it is a big issue. It's a bacterial enzyme, so highly immunogenic. There are really 2 uses, and we think it's a great fit for our platform. Our primary focus is to use the IgG protease to pretreat patients with pre-existing antibodies, so had a natural AAV infection, to expand the patient pool.

But there is a second application in autoimmune disease. As you rightly said, there is a commercial product, an IgG protease in Europe, that is limited to a single dose due to the immunogenicity. And I think we have the potential to re-dose Xork, and we opened up 2 more indications. And specifically, there are a number of IgG-mediated autoimmune diseases where we can apply Xork.

Our next question will come from Yun Zhong with BTIG .

This is Xu on for Yun Zhong. So I have a couple of questions regarding your MMA program. So first is about the CMC review by the FDA. Can you give me -- us more color? Is that because FDA needs more time to review? Or you kind of foresee FDA may request more data?

And also, when do you think you will give us an update on the clinical design or MMA? I know your Phase I study will explore ImmTOR's first-dose [strategies]. So do you think, on your Phase I study, you may like include the re-dosing strategies in those patients, if needed?

Yes. Thanks for the question, Xu. So we -- unfortunately, we can't provide a lot more color. We don't have more color. We, as you can read in our filings, the FDA 30-day review period expired. We're orally communicated by the FDA that they're still considering certain aspects of the submission around CMC, and then we'll get a formal decision by the end of November.

So unfortunately, we also don't have more color around when do we have guidance on the clinical design. Obviously, once we have IND approval, we'll share the clinical trial design in detail and also some of the endpoints we're going to look at in the Phase I trial.

Great. I may ask one more question here. So just some competitive landscape in MMA. Just for a very few or very rare indication, the space can be quite crowded. Just name a few, like the large bios, AAV gene therapy and Moderna have MRA program and a few other auto solutions in trials. So I mean, it's great for the patient community.

But from a business perspective, I just want to know your thoughts on this. Is that because the disease may be like escalating underdiagnosed. There may be more patients than estimated, or is a good indication for proof of concept? And also, do you participate -- anticipate some like patient enrollment challenge?

Yes, that's a good question. We obviously think that we do have a highly differentiated approach here with the potential to re-dose. No other approach can offer that. So we think we're quite differentiated from that perspective. It is still -- it's not an ultra-rare indication. It's a rare indication. So you're looking in the thousands of patients.

And we've been very strategic about who we work with. So we have a partnership with the NIH, with Chuck Venditti, who is one the permanent researchers in the field. And they maintain the largest patient registry in the U.S., so we'll have access to those patients. So we've been mindful around partnering with someone that has access to MMA patients.

Our next question will come from Ram Selvaraju with H.C. Wainright.

This is [Amaz] on for Ram. Thanks for the comprehensive update. So 3 from us, if I may. So enzyme replacement therapy in the context of lysosomal storage diseases is kind of like a foundational treatment. From your perspective, we were wondering what are some of the drawbacks of ERT in this context? And how are you proposing to replace the existing treatment paradigms through ImmTOR?

Yes. I'll let Peter answer that in more detail. But obviously, ERTs are limited in their use. They're easily highly immunogenic enzymes. And if you look at the overall response rates, are not as high as -- whereas, obviously, gene therapy, you're addressing the underlying monogenic issue -- pathology. But I'll let Peter provide a bit more color here.

Yes, Carsten, I think that you've hit the key issue with enzyme replacement therapy, you have to give it repeatedly. And they are immunogenic compounds, so you develop antidrug antibodies, which then reduces the efficacy. Of course, adding ImmTOR to ERTs could potentially improve that. But with our partnerships, particularly with Takeda, we have taken a different approach, and that is to team up with gene therapy because gene therapy will likely reduce the long-term nature of the ERT and give you more long-term treatment.

And furthermore, if we add ImmTOR in that, if it does wane an expression, we can potentially do repeat dosing of the gene therapy. Additionally, ImmTOR with the gene therapy could also prevent antibodies to the -- express transgene. So there are a couple of ways that ImmTOR, combined with gene therapy, is the approach that we're pursuing rather than ERT.

Shifting on to the partnership with Ginkgo. Are you able to expand on the logistics of bringing ImmTOR together with their foundry platform? For example, will ImmTOR capability be installed at the foundry? Or will the platforms operate independently in their current locations?

Yes. So they'll operate independently, and we're really excited about the partnership with Ginkgo. And we're basically using their foundry to develop novel and next-generation enzymes, which we then plan to combine with ImmTOR. So what's important is that we will have exclusive therapeutic use for those enzymes. So in a sense, it's a strategic partnership, but it's really an inbound deal where we get access to some of their novel enzymes that we plan to combine with ImmTOR to enable re-dosing.

Fantastic. And then just finally, are you able to reveal specific design capabilities of Ginkgo's foundry platform that you think will enhance the sustained efficacy of biologics with ImmTOR technology? And in addition, if you're able to reveal some initial disease indications that you're pursuing with Ginkgo, that would be great, too.

Yes. So we haven't disclosed details, we just gave kind of the broad brush. We're pursuing autoimmune diseases with an enzymatic therapy. And maybe I'll let Kei talk a little bit about their technical capabilities, but what really struck me is the quality and the speed they can identify enzymes and modify enzymes. It really convinced me that they're in quite a unique position in the landscape of protein design, protein engineering companies. But maybe, Kei, do provide a little more color.

Yes. So yes, obviously, Ginkgo has made quite an impact in the protein engineering world. I think their foundry technology really industrializes protein design and testing. So I think, as Carsten mentioned, we're really excited to be working with them. And I think, at a future date, we'll be able to provide you more information on our partnership.

Thank you. This concludes our question-and-answer portion of the call. I will now turn the call back over to Selecta's CEO, Carsten Brunn, for any closing remarks. Carsten?

Yes. Thank you, operator, and thank you, everyone, for joining us this morning. Stay safe and healthy. And this concludes today's call. Thank you.

The conference has now concluded. Thank you for attending today's presentation. You may now disconnect.