Taysha Gene Therapies, Inc. Q2 FY2025 Earnings Call

Ladies and gentlemen, good morning, and welcome to Taysha Gene Therapy's Second Quarter 2025 Earnings Conference Call. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Haleigh Collins, Director, Head of Corporate Communications. Please go ahead.

Thank you. Good morning, and welcome to our second quarter 2025 financial results and corporate update conference call. Earlier today, Taysha issued a press release announcing financial results for the second quarter ended June 30, 2025. A copy of this press release is available on the company's website and through our SEC filings. Joining me on today's call are Sean Nolan, Taysha's Chief Executive Officer; Sukumar Nagendran, President and Head of R&D; Kamran Alam, Chief Financial Officer; and Dr. Elsa Rossignol, Director of the Integrated Rett Syndrome Clinic at St. Justine in Montreal and principal investigator of the REVEAL Phase I/II trials. We will be presenting slides to accompany our prepared remarks today, which will be available on our website after the call. We will host a question-and-answer session following our prepared remarks. Please note that Dr. Rossignol will be available to take questions until 9:20 a.m. Eastern Time, after which she will be stepping away for her clinic commitments. On today's call, we will be making forward-looking statements, including statements concerning the potential of TSHA-102, including the reproducibility and durability of any favorable results initially seen in patients dosed to date in clinical trials, including with respect to functional milestones and to our other product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, including the timing of initiating additional trials, reporting data from our clinical trials and making regulatory submissions; timing or outcomes of communications with the FDA in Health Canada on the regulatory pathway for TSHA-102 the potential for product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies and the market opportunity for our programs. This call may also contain forward-looking statements relating to Taysha's growth, forecasted cash runway and future operating results, discovery and development of product candidates, strategic alliances and intellectual property as well as matters that are not historical facts or information. Various risks may cause Taysha's actual results to differ materially from those stated or implied in such forward-looking statements. For a list and description of the risks and uncertainties we face, please see the reports we filed with the SEC, including in our annual report on Form 10-K for the full year ended December 31, 2024, that we filed February 26, 2025. Our quarterly report on Form 10-Q for the quarter ended June 30, 2025, we filed today. This conference call contains time-sensitive information that's accurate only as of the date of this live broadcast, August 12, 2025. Taysha undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date of this call, except as may be required by applicable securities laws. With that, I would now like to turn the call over to our CEO, Sean Nolan.

Thank you, Hayleigh, and welcome, everyone, to our second quarter conference call. I will begin with an update on our recent activities and regulatory progress for our lead TSHA-102 Rett syndrome program, including new details of our FDA-aligned pivotal trial design and our registrational pathway. Next, Suku will discuss our natural history data analysis, which underpins our REVEAL pivotal trial design. I've invited Dr. Elsa Rossignol, Director of the Integrated Rett Syndrome Clinic at St. Justine in Montreal and a principal investigator of the REVEAL trials, to present the previously disclosed Part A clinical data from our REVEAL Phase I/II trials that she presented at the International Rett Syndrome Foundation Scientific Meeting in June. Those who attended may recall how impactful her presentation was to the audience. Today, we're excited to expand its reach to the broader community as these data have been central to our clinical discussions with regulators in preparation for our pivotal trial. Kamran will then follow up with a financial update, and I will provide closing remarks before opening the call to questions. We have continued to make strong progress supporting the advancement of our TSHA-102 program. This included obtaining alignment with the FDA and Health Canada to proceed with initiating our REVEAL pivotal trial, reporting positive clinical data supporting the therapeutic potential of TSHA-102 and strengthening our balance sheet. We believe this meaningful progress sets us on a clear and efficient path toward the potential registration of TSHA-102. In May, we announced that we had reached alignment with the FDA on key design elements of our REVEAL pivotal trial and next steps for enabling study initiation. Subsequently, we submitted our IND application amendment to the FDA and CTA amendment to Health Canada. I am now pleased to report that we have officially commenced the site activation for our REVEAL pivotal trial in accordance with the key design elements we previously aligned on with the FDA following receipt of the no objection letter from Health Canada and feedback from the FDA. As a result of this progress, we anticipate beginning patient enrollment for our pivotal trial in the fourth quarter of this year. Our frequent and constructive dialogue with the FDA through the RMAT mechanism has been instrumental in enabling us to navigate this novel regulatory pathway, which I will discuss in more detail shortly. Rett syndrome is a rare, progressive, and debilitating neurodevelopmental disease affecting an estimated 15,000 to 20,000 patients across the U.S., Europe, and U.K. It often necessitates 24/7 care and lifelong support. Despite the severity, there are no currently approved therapies that treat the underlying genetic root cause of this disease, underscoring the urgency for new therapeutic advancements. TSHA-102 is a highly differentiated gene therapy candidate designed to target the genetic root cause of Rett syndrome. With key attributes that intend to support safety and potential commercial viability, we believe TSHA-102 is poised to redefine the treatment landscape for Rett syndrome. Specifically, it leverages the clinically and commercially proven AAV9 vector, which has a well-characterized safety profile demonstrated in studies for other third-party gene therapies across multiple indications. Another important distinction is that TSHA-102 is administered via lumbar intrathecal injection, which is a routine minimally invasive delivery approach. Commercially, this can be advantageous in that it does not require a surgical suite or expert neurosurgery delivery and can be performed as a routine outpatient procedure. Additionally, intrathecal administration delivers the vector directly to the cerebrospinal fluid, facilitating widespread biodistribution and transduction within the CNS while limiting systemic circulation. This reduces peripheral tissue exposure that may help lower the risk of off-target effects, including immune responses and systemic toxicities, thereby potentially contributing to a more favorable safety profile. From the outset, our clinical development strategy has been deeply data-driven with a focus on defining the most objective, clinically meaningful way to assess TSHA-102 across a broad patient population. Our robust analysis of the Rett syndrome natural history data set demonstrated that after the age of 6 years, the likelihood of achieving defined developmental milestones across the core functional domains of Rett syndrome is approximately 0%. This established a developmental plateau population. These important findings underpin our FDA-aligned pivotal trial design and allow us to objectively measure the functional aspects of TSHA-102 on essential activities of daily living. As I mentioned, we are pleased to have commenced site activation for our REVEAL pivotal trial, which will assess the percentage of patients in the developmental plateau population who gain or regain one or more developmental milestones as part of the primary endpoint, with each patient serving as their own control. Based on the Part A data from our REVEAL 1/2 trials, it's encouraging that all 10 patients treated with TSHA-102 gained or regained one or more developmental milestones, corresponding to a 100% response rate for the pivotal trial primary endpoint based on the May 25 data cutoff. With safety at the forefront, we are pleased to share that the low and high doses of TSHA-102 continue to be generally well tolerated with no treatment-related serious adverse events or dose-limiting toxicities in the 12 patients treated as of the August 2025 data cutoff. Lastly, we recently completed a public follow-on offering that resulted in total gross proceeds of $230 million, including full exercise of the underwriter's option to purchase additional shares, extending our cash runway into 2028. With our balance sheet strengthened, our pivotal trial underway, and a defined path to registration, we believe we are well positioned to advance TSHA-102 to benefit patients living with this devastating disease. Our REVEAL clinical development program was designed to support the future approval of TSHA-102 for the treatment of females aged 2 years and older with Rett syndrome. Recall, Part A was our dose escalation phase where we treated 12 patients aged 6 to 21 years in our 2 Phase I/II REVEAL trials with one of the two dose levels. The totality of the Part A data helped inform our discussions with the FDA and Health Canada on the optimal trial design for Part B, the pivotal phase of our trials. Our REVEAL pivotal Part B trial will evaluate the gain and regain of developmental milestones in the developmental plateau population. In parallel, we previously announced we are aligned with the FDA on an extrapolation approach in a separate safety-focused study evaluating TSHA-102 in the pre-developmental plateau population of females aged 2 to less than 6 years. Given the high incidence of spontaneous milestone gains in this population, safety will be the primary focus, and efficacy will be extrapolated from the developmental plateau population. Importantly, we believe this two-study approach allows us to generate safety and efficacy data across the broad Rett syndrome population while mitigating disease heterogeneity risk. Leveraging a pivotal trial design focused on targeted enrollment of patients within the developmental plateau population provides high statistical confidence given the approximately 0% likelihood of spontaneous milestone achievement in this population. From a CMC perspective, the pivotal TSHA-102 product has been released and cleared for use in our REVEAL pivotal trial. As previously disclosed, the FDA approved the use of the pivotal lot, which is manufactured from the planned commercial manufacturing process and agreed that it is comparable with the clinical material used in Part A. This achievement supports product consistency and quality, which are essential pillars of safety. Furthermore, this streamlines our path to initiating the pivotal trial and underscores our CMC readiness to support a future BLA submission. Our REVEAL pivotal trial for TSHA-102 will reflect a single-arm open-label pivotal trial design with each patient serving as their own control. The high dose of TSHA-102 of 1E of the 15 total vector genomes will be evaluated, and we will enroll 15 females between the ages of 6 and less than 22 years in the developmental plateau population. As mentioned, the primary endpoint will assess the percentage of patients who gain or regain one or more developmental milestones from the list of 28 milestones across the core functional domains of communication, fine motor, and gross motor. Importantly, this responder definition was supported by the FDA and Health Canada as part of our recent feedback. With this established, we continue to believe Part A of our REVEAL trial supports the pivotal trial is well powered to establish efficacy. We plan to conduct a 6-month interim analysis in addition to a 12-month primary analysis, which could potentially expedite BLA submission by 2 to 3 quarters. Key secondary endpoints include the average number of total developmental milestones gained or regained per patient following TSHA-102 as well as clinician-assessed outcomes, including the RMA and the CGI-I. These secondary endpoints are designed to capture the broad therapeutic outcomes across the domains of communication, fine motor, gross motor, and autonomic function that are central to the burden of disease. We are focused on site activation, which we anticipate will enable us to begin patient enrollment in the fourth quarter of this year. Importantly, our pivotal trial primary endpoint is an objective, clinically meaningful, and individualized assessment of function in the developmental plateau population. It was supported by caregiver research and natural history and has been aligned with the FDA. As such, we believe achievement of this endpoint has the potential to redefine expectations and expand the possibilities of gene therapy for patients with Rett syndrome. With the pivotal trial now underway, it is important to understand the data-driven approach we took to defining this novel regulatory pathway, which was informed by the natural history and our REVEAL Part A clinical data. I will now turn the call over to Suku to discuss this. Suku?

Thank you, Sean. The longitudinal NIH-funded IRSF natural history study data represents the largest global Rett syndrome natural history data set in which patients were assessed by direct examination. This included longitudinal data on the gain, loss, and regain of developmental milestones in the co-functional domains of Rett syndrome. These data were collected via clinician conducted interviews with primary caregivers at 6- to 12-month intervals. We selected a large cohort of approximately 1,100 females with up to 14 years of follow-up who closely aligned with our REVEAL trial enrollment criteria. We use this data to build age and time-based cumulative incidence curves, which demonstrate the likelihood of gaining a new milestone based on age and the likelihood of regaining a milestone that was lost based on time since loss. These models showed clear age and time-based trends in developmental milestone acquisition that have strengthened the field's understanding of longitudinal disease progression, substantiated the disease-modifying potential of TSHA-102, and informed our discussions with the FDA on our proposed pivotal trial design. As seen on the slide, we built a tool that predicts the likelihood of a developmental milestone gain based on age and regain based on time since loss. Importantly, these incidence models demonstrated that the likelihood of gaining or regaining any of the 28 defined developmental milestones for our primary endpoint is highly predictable in patients 6 years of age or older. Here, you will see 3 examples of milestone gains across the 3 core domains, demonstrating that when an untreated patient achieved these milestones, it occurred before the age of 6. After the age of 6, the curve flattens with approximately 0% likelihood of an individual gaining these milestones. We leverage these findings to establish the developmental plateau population, which is the population of age 6 years and older, at which the likelihood of milestone gain and regain becomes predictable. This slide highlights the 28 developmental milestones selected for inclusion in our primary endpoint, each meeting predefined criteria, that is, they are meaningful to caregivers, represent activities of daily living, and demonstrated between 0 and less than 6% likelihood of spontaneous achievement in untreated individuals aged 6 years and older. This illustrative graphic helps put these findings into context. Our natural history analysis uncovered the developmental plateau population of patients aged 6 years and older, where there's an exceedingly low chance of gaining new developmental milestones and regaining developmental milestones that were lost after a defined number of years. Therefore, the gain of new or restoration of previously lost milestones presents an objective, clinically meaningful, and data-driven way to assess the efficacy of TSHA-102 in a broad population. We applied rigorous evaluation criteria to our Part A developmental milestone data to enable a reliable, objective, and consistent assessment of TSHA-102's efficacy. As a first-in-human study, we evaluated a range of clinical outcome assessments in Part A to generate a robust data set to inform our pivotal trial design. As such, select developmental milestones were captured through video recorded evaluations, clinical outcome assessments, caregiver assessments, and clinician notes. We applied structured evaluation criteria to the available video evidence milestones. To qualify as a milestone achievement following TSHA-102, there were 3 criteria that had to be met. First, the milestones have either never been gained or were lost sufficiently long ago such that the likelihood of spontaneous gain or regain was less than 6.7% based on reviews of patients' medical history and available baseline video data. Second, there had to be video documentation of the milestone achievement post TSHA-102 treatment. Third, this video evidence was independently evaluated by multiple external central raters who used prespecified definitions of achievement for each of the 28 milestones based on the pivotal trial protocol to determine whether a milestone was demonstrated. By leveraging this criteria, we believe the Part A of our REVEAL trials is a reliable reflection of TSHA-102's impact on developmental milestone achievement well beyond what's statistically probable for these patients. Importantly, our pivotal trial is well powered to establish the therapeutic impact of TSHA-102. Beyond the video evidence milestone data, we collected data that further demonstrate developmental milestone achievements and functional improvements in activities of daily living across the core domains of the disease, which we look forward to sharing in the fourth quarter of this year to further support the broad therapeutic impact. With that, I'm pleased to turn the call over to Dr. Elsa Rossignol to present the previously disclosed Part A data. Elsa?

Thank you. It's a pleasure to be here and to describe what we presented in the IRSF meeting in June. Next slide, please. As we move on, we've observed what Suku mentioned. Can you move the slide? Are you hearing me?

Yes, we can hear you.

Okay. Very good. So please move the slides. Thank you. All right. So as Suku was mentioning, the data from Part A was reanalyzed using independent central raters to assess gains or regains of any developmental milestones that were part of these 28 milestones isolated based on the natural history study data. And all of these milestones, I remind you, are typically never gained or regained past the age of 6. The data that we currently had in the trial was reanalyzed based on video. So, it was a very strict detection of gain or regain using predefined primary criteria. We were very pleased to see that all patients treated so far in the cohort have reached this criteria of gaining at least one milestone or regaining at least one milestone, and many patients actually gained more than one milestone as you will observe in the next slide. Before we go on, I just want to point out that in the data we're presenting here for the clinical assessments, we're describing 10 patients. The 2 other patients were below the 3-month cutoff, so we didn't have yet the clinical assessments to detect and conduct this evaluation. I also want to point out that, when you look at the data, of course, the longer follow-up were for the low-dose cohort. For the high-dose cohorts, we have a shorter follow-up, and many of them were still adolescent. We are eager to see the development of this data moving forward with the 12 months and 18 months cutoff for the high-dose patients. Looking at the gains that we've observed in the cohort so far, out of the 10 patients that were treated, next slide. Out of the 10 patients that were treated, we've observed a gain of 22 developmental milestones, and these were across multiple domains of the disease as illustrated here. Patients that gained milestones in one domain often gained also in other domains, so it's not restricted to one single domain. 22 milestones out of 10 patients suggest that many patients gain more than one. The domains that we've observed with gains or regains are, for instance, in communication. We saw patients that are now able to use phrases to communicate things like, okay, bye, where before they would use sparse words here and there or one single word very infrequently. Many patients also gained the ability to follow commands with a gesture or to identify body parts. This may seem like just a basic task for anyone who has typical development, but for patients with Rett, this is critical because they've finally been able to point to what they really want. Communication was key, both receptive and expressive. In terms of fine motor, we've seen patients gaining the ability to hold a bottle on prop, a real gain in autonomy and independence. In terms of gross motor scales, we've seen patients starting to be able to walk with support. All of these show real gains in terms of independence and mobility, and it reduces the physical burden on caregivers.

Thank you, Dr. Rossignol. Research and development expenses were $20.1 million for the 3 months ended June 30, 2025, compared to $15.1 million for the 3 months ended June 30, 2024. The $5 million increase was driven by BLA enabling process performance qualification manufacturing initiatives, REVEAL clinical trial activities, and higher compensation expenses as a result of increased headcount during the 3 months ended June 30, 2025. General and administrative expenses were $8.6 million for the 3 months ended June 30, 2025, compared to $7.3 million for the 3 months ended June 30, 2024. The increase of $1.3 million is primarily due to higher legal and professional fees. Net loss for the 3 months ended June 30, 2025, was $26.9 million, or $0.09 per share, compared to a net loss of $20.9 million, or $0.09 per share for the 3 months ended June 30, 2024. As of June 30, 2025, Taysha had $312.8 million in cash and cash equivalents. This reflects gross proceeds of $230 million from the May 2025 follow-on financing, which includes the full exercise of the underwriters' option to purchase additional shares. We also refinanced our existing loan and security agreement with Trinity Capital from the original $40 million, which was paid in full, to a new debt facility equal to $50 million upfront. The refinanced loan defers debt principal payments by more than 2.5 years, lowers our interest rate, and provides access to nondilutive capital. Importantly, there continue to be no financial liquidity covenants or warrants associated with the new refinance loan with Trinity. We look forward to our continued partnership with the Trinity Capital team. We expect our current cash resources to support planned operating expenses and capital requirements into 2028. I will now turn the call over to Sean for his closing remarks.

Thanks, Kamran. I'm pleased with the progress we have continued to make to advance our TSHA-102 program. With a strengthened balance sheet, our FDA-aligned pivotal trial underway, and compelling clinical data from Part A, we are moving forward with confidence as we work to deliver on our anticipated near-term milestones. This includes beginning patient enrollment for our pivotal trial in the fourth quarter of this year. We also plan to report new supplemental clinical data from Part A of our REVEAL Phase I/II trials supporting the broad therapeutic impacts of TSHA-102 in the fourth quarter of this year. We truly appreciate the collaborative interactions with the FDA in Health Canada to date and believe this progress advances our goal to bring TSHA-102 to patients with this devastating disease as expeditiously and safely as possible. I will now ask the operator to begin our Q&A session.

We take the first question from Salveen Richter from Goldman Sachs.

This is Lydia on for Salveen. I guess given there was a 100% response rate for the pivotal trial primary endpoint in the Part A of the study, is this the bar for Part B in your view?

I think that the results we've seen in Part A are exceptionally compelling. I think 100% is always a difficult standard to keep. I think what's the most important thing is how we built the statistical plan for Part B. And keep in mind that we're using a null hypothesis of 6.7%, which was the highest percentage of a gain or regain per milestone that we're evaluating. When you take that into account with a 15-patient population, that would mean 1 patient out of 15 would spontaneously have a potential gain or regain. The statistical bar to get over is relatively low. All things being equal, it's approximately 33% based on our submission of the SAP. What gives us a lot of confidence is that the numbers that we're seeing so far play out in Part A are consistent and significantly above that threshold. Thank you.

I have a question for Dr. Rossignol. It was encouraging to hear about the reported milestones that weren't part of the video assessments. Can you help us understand if there is a specific time frame for when all these milestones are occurring? Are you observing additional milestones happening over time? I’m trying to determine if you believe the benefits of the gene therapy have reached a plateau.

Yes. Thank you for the question. If you look at the data that we have so far up to the cutoff, we were seeing increasing improvement as we were following the patients. And so we're expecting a similar increase in gains over the time course since that last cutoff. This does not occur just in one visit, and we do see gains usually at most visits in follow-up. When we mentioned the gains that are not observed or quantified in this developmental milestone assessment, what I meant by that is if you're now able to walk with a walker, that doesn't give you a point on the developmental milestone scales because you would need to walk independently. We can have critical gains that improve daily functioning that are not captured in this skill set but are still major and critical improvements.

I wanted to maybe talk about the differentiation of your gene therapy versus your competitor. In particular, maybe I could get the doctor's view on study design. So you're looking at a smaller cohort and you're also looking at a single primary endpoint. Can you talk about the differences in these studies and how you think this could lead to differentiation between the 2 programs?

Yes. Thanks, Tazeen. It's good to hear from you. Suku, maybe we can tag team this. I think first and foremost is the fact that the way we conducted the Part A trial was to cast a broad net. It was really the first-in-human trial to let us explore what are some of the different efficacy measures that would make a lot of sense. We've always been of the mind that with gene therapy, you really have to show something clinically meaningful and objective that's going to impact activities of daily living, i.e., you can go back 2 years and hear Suku talk about small gains and scales are not going to be the mark that we're looking for. The fact that we've been working with the regulators to establish a completely new paradigm in assessing clinical efficacy, which is the gain and regain of milestones, is functional gains that have never been achieved before in this disease. We're redefining how we approach defining what truly is impactful from a clinical perspective and from a patient and caregiver perspective. The additional endpoints that we're looking at relative to RMBA, CGI, again, we can benchmark RMBA to the natural history, and it's exceptionally compelling. Natural history is about 0, right? It just doesn't move. We're showing that it's better. That's going to help with the excitement in the community, and that's going to help with the payers and supporting them. Combine the efficacy impact with the safety we've been able to generate to date, we think that this is a very enticing offer to patients. They are going to want to do what's easiest, safest, and that demonstrates a significant effect that can change their daily life.

Sean, I would like to add one more point. I want to reiterate that at Taysha, we are fully dedicated to patients with Rett syndrome. We demonstrate this commitment by focusing on the safety and clinical significance of our product through a minimally invasive administration method. Regarding our competitor's protocol, for transparency, I am not aware of their protocol beyond what is publicly available. It seems to be a single-arm study that had a composite endpoint for the primary outcome; however, it remains unclear if that is still the case based on recent disclosures. Our natural history data set is extensive and allows for a thorough assessment over a 14-year timeframe. When looking at patients over the age of 6 who receive a therapeutic intervention like TSHA-102, if they achieve a new skill or regain a previously lost ability, the evidence strongly suggests it is due to Taysha's gene therapy. We have observed patients transforming their lives by acquiring these skills and enhancing their daily living activities.

This is Ethan on for Gil. Congratulations on the progress this quarter. Your competitor recently received regulatory feedback that a 6-month endpoint would not be considered clinically meaningful in their case. Obviously, your study designs are slightly different, but would you expect any pushback on using your interim readout to support BLA filing? Or was that already discussed with the FDA as well?

Yes. It was part of our submission. We've taken a very data-driven approach. When you think about what underlies our interim analysis approach, it's based on the proof-of-concept analysis of the Part A data, which provides robust support for the primary endpoint at both 6 and 12 months. At the high dose, there was an 83% responder rate at 6 months, and that improves and deepens over the course of 12 months. We have not been pushed off the ball about doing a 6-month interim. It's more the particulars of the assessments. We feel good about where we stand at this particular point in time.

This is James on for Maury. Congratulations on the progress this quarter. Can you talk about expectations for the new supplemental REVEAL Part A data in Q4? Could we get additional data points like more granular information on the number of video documented milestones gained across the high and the low dose or individual CGII data points? Do you anticipate that the data will be at a medical conference or a company update?

I can see it being at both a medical conference and a company update. We'll provide more specifics over the coming weeks and months leading into it, James. There's a lot of information that we captured, and we cast a wide net. Some of that net also included additional videos that we haven't talked about, different ways to capture milestone gain. We want to share with the community additional milestones and what those look like, how those translate into activities of daily living, and improvements in the quality of life of the patients that have been treated thus far.

Can you maybe characterize any changes from the Part A CMC material batch to Part B CMC material with a specific emphasis on full empty capsid ratio across both batches? With the commercial scale of material, what are your thoughts in terms of how many patients you can supply into the market when approval comes in?

Yes, Biren. It's a massive opportunity. If we're talking around 10,000 patients in the U.S., it's an immense unmet need. We have an excellent leader, Fred Porter, leading the effort to make the highest quality product and to scale it. We aligned with the FDA that the Part A and pivotal material are analytically comparable, which is very important. We've made the product for the pivotal trial; that’s made with the commercial process at scale. We've sent that to the FDA. We could have extended our cash runway into mid-2028 because of our raise, but we opted to keep the guidance into 2028, to begin building the commercial inventory we need. We've got that as a contingency, and we feel strongly that we're in a good position on the CMC side at this point.

I wanted to ask just more specifically on your recent interactions with regulators. It seems like you have sign-off from the Canadian authorities. But I wanted to understand where it sits as it relates to the IND amendment with the FDA and any ongoing discussions there. What are the points of conversation as it relates to those conversations, if they're ongoing, or have you achieved a formal signoff? Where do things sit there?

Yes, Jack, great question. There’s a difference in process. We received a no objection letter from Health Canada on day 37 with no comments, meaning you're good to go. In the U.S., with the FDA, because we did an IND amendment, there's no official correspondence. There’s generally been an unofficial 30-day if you don’t hear something, you can proceed. We were told early on in July that there were no clinical hold concerns. Our view is that the ongoing FDA dialogue is focusing on how to position the program for success versus overturning key trial design elements, so it’s been constructive and supportive.

Ladies and gentlemen, we take that as the last question and conclude the question-and-answer session. I will now hand the conference over to Mr. Sean Nolan for his closing comments.

Just want to thank everyone for their time this morning, including our special guest, Dr. Elsa Rossignol, and we wish you all a great week. Take care, and we'll talk soon.

Thank you. Ladies and gentlemen, the conference of Taysha Gene Therapies has now concluded. Thank you for your participation. You may now disconnect your lines.