Key Takeaways FDA grants platform tech designation to SRPT's rAAVrh74 viral vector used in SRP-9003 The tag lets Sarepta reuse clinical and manufacturing data across other rAAVrh74-based therapies. SRP-9003 is in a phase III trial for LGMD2E/R4; data expected later this month.Sarepta Therapeutics (SRPT) announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, used in the investigational gene therapy SRP-9003. This therapy is being evaluated in a late-stage study for ...

RGX-202: AFFINITY DUCHENNE® New Phase I/II Interim Functional Data June 2025 Forward-looking statements This presentation includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," " ...

Core Insights - REGENXBIO Inc. announced positive interim data from the Phase I/II AFFINITY DUCHENNE trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, showing consistent functional benefits and safety [1][2][3] Functional Data - RGX-202 participants at dose level 2 (2x10^14 GC/kg) showed significant functional improvements at both 9 and 12 months, exceeding natural history controls on key measures such as the North Star Ambulatory Assessment (NSAA) [4][5] - At 9 months, participants improved an average of 4 points from baseline on NSAA, and at 12 months, the improvement was 4.5 points from baseline, with a 6.8-point increase compared to natural history [5][6] Biomarker Data - Biomarker data indicated robust microdystrophin expression, with one participant aged 2 showing an expression level of 118.6% compared to control [8][10] - The primary endpoint for the pivotal phase is the proportion of participants with microdystrophin expression ≥10% at Week 12 [8] Safety and Tolerability - RGX-202 demonstrated a favorable safety profile with no serious adverse events reported, and common drug-related adverse events included nausea, vomiting, and fatigue [11][12] - The proactive immune modulation regimen and high product purity levels contributed to the positive safety outcomes [11] Pivotal Trial and Future Plans - REGENXBIO is enrolling participants for the pivotal portion of the AFFINITY DUCHENNE trial, aiming to support a Biologics License Application (BLA) submission under accelerated approval by mid-2026 [14][15] - The company plans to share top-line data in the first half of 2026, including biomarker, functional, and safety data [15] About RGX-202 - RGX-202 is designed to improve function and outcomes in Duchenne muscular dystrophy, utilizing a differentiated microdystrophin construct that encodes key regions of dystrophin [17][18] - The therapy aims to support targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV® AAV8 vector [18] About Duchenne Muscular Dystrophy - Duchenne muscular dystrophy is a severe, progressive muscle disease affecting 1 in 3,500 to 5,000 boys born each year, caused by mutations in the dystrophin gene [19] About REGENXBIO Inc. - REGENXBIO is a biotechnology company focused on gene therapy, with a late-stage pipeline including RGX-202 for Duchenne and other investigational therapies for rare diseases [20]

Core Viewpoint - Ocugen, Inc. has signed a binding term sheet to negotiate a licensing agreement for exclusive rights to its gene therapy OCU400 in Korea, aimed at treating retinitis pigmentosa (RP) [1][4]. Group 1: Licensing Agreement Details - The licensing agreement will provide Ocugen with upfront fees and near-term development milestones totaling up to $11 million [2][8]. - Ocugen will receive sales milestones of $1 million for every $15 million in net sales in Korea, along with a royalty of 25% on net sales generated by its partner [2][8]. - The company will manufacture the commercial supply of OCU400 under a supply agreement [2]. Group 2: Market Opportunity - There are approximately 15,000 individuals in South Korea affected by RP, presenting a significant market opportunity for the partner to lead in gene therapy [3]. - The regional licensing strategy aligns with Ocugen's goal to partner with established companies to maximize patient reach while generating returns for shareholders [4]. Group 3: Development Timeline - Ocugen is advancing OCU400 through Phase 3 clinical development, with a target for filing a Biologics License Application by mid-2026 [5].

Summary of Conference Call Company Overview - **Company**: 4D Molecular Therapeutix (4D MT) - **Lead Product**: 4,150 for wet Age-related Macular Degeneration (AMD) and Diabetic Macular Edema (DME) Industry Context - **Market Size**: The market for retinal vascular diseases is estimated to reach **$20 billion** by the time the product is launched [3] - **Current Treatments**: The current treatment landscape includes EYLEA and Vabismo, with a focus on improving durability and reducing treatment burden [29][30] Key Product Updates - **Clinical Trials**: The Forefront I Phase III trial is currently underway with over **50 clinical trial sites** activated, targeting a total of **100 sites** [2] - **Product Designation**: 4,150 is the first known genetic medicine to receive RMAT designation, indicating its potential as a breakthrough therapy for wet AMD and DME [3][46] - **Efficacy**: In Phase 1/2 trials, 4,150 demonstrated an **83% reduction** in injection burden for hard-to-treat patients, with some recently diagnosed patients showing over **90% reduction** [12][13] Core Product Advantages - **Durability and Safety**: The product aims to provide greater durability with a favorable safety profile, comparable to EYLEA [4][15] - **Treatment Burden Reduction**: The anticipated reduction in treatment burden is projected to be between **80% to 90%**, significantly higher than existing therapies [8][30] - **Patient Convenience**: The product is designed for seamless integration into clinical practice, allowing for routine intravitreal injections [4] Market Dynamics - **Physician Feedback**: There is significant interest from physicians and patients regarding gene therapy, with a focus on durability and treatment burden reduction [30][31] - **Payer Environment**: The majority of wet AMD patients are Medicare patients, which is seen as a favorable payer environment for launching the product [43] Regulatory Insights - **FDA Alignment**: The FDA has agreed that a single Phase III trial can serve as the basis for BLA submission for DME, which is a significant regulatory milestone [51] - **RMAT Designation**: This designation is expected to facilitate more efficient interactions with regulatory bodies and potentially streamline the review process [46][47] Financial Considerations - **Cost of Goods**: The cost of goods for 4,150 is projected to be less than **$1,000**, providing flexibility in pricing strategies [44] - **Funding for DME Trial**: The company is exploring non-equity financing and partnerships for funding the DME trial, with current cash reserves sufficient to support wet AMD programs through data readout [53] Future Milestones - **Upcoming Data Releases**: Key data updates are expected in Q4 for Phase 1/2 trials, with continued updates on enrollment and site openings for Phase III trials [61] - **Cystic Fibrosis Program**: The company is also advancing its 4,710 product for cystic fibrosis, with updates expected in the second half of the year [25][59] Conclusion 4D Molecular Therapeutix is positioned to potentially revolutionize the treatment landscape for wet AMD and DME with its innovative gene therapy approach, focusing on significant reductions in treatment burden and improved patient outcomes. The company is actively engaging with regulatory bodies and exploring funding options to support its clinical programs.

Summary of Abeona Therapeutics (ABEO) Conference Call Company Overview - Abeona Therapeutics is a commercial stage cell and gene therapy company that recently received approval for ZivaSkin, a therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB) [5][70] - The company has a market cap of approximately $300 million and anticipates becoming profitable in the early half of 2026 [6][71] Key Developments - ZivaSkin was approved just over a month ago, and the company has entered into a sale agreement for a Priority Review Voucher (PRV) for $155 million [5][70] - Abeona is funded for eight quarters of operations without accounting for revenue generation [6][71] Launch Metrics and Treatment Centers - Lurie Children's Hospital has been activated as the first qualified treatment center for ZivaSkin [7][72] - The company is tracking patient throughput and referral volume from other centers of excellence [7][72] - The timeline to fully activate all five treatment centers is by the end of 2025 [9][74] Patient Demand and Treatment Capacity - There are currently an upper double-digit number of patients eligible for treatment across the five centers [11][73] - The company expects to treat 10 to 14 patients this year, with the capacity to ramp up to 10 patients per month by the first half of 2026 [18][37] - The treatment window runs from August to November, with minimal disruption expected due to a planned December manufacturing shutdown [20][21] Pricing and Payer Engagement - ZivaSkin is priced at $3.1 million, which is considered reasonable compared to the standard of care costs [28][30] - The company has not faced pushback from payers and has entered agreements covering over 100 million commercially insured lives [31][36] Financial Outlook - Abeona expects to achieve cash flow positivity in 2026 with a target of treating 36 patients [37][39] - The company is confident in exceeding this target, potentially treating up to 50 patients [38][39] Future Plans and Market Expansion - Abeona is exploring regulatory submissions outside the U.S. and assessing reimbursement scenarios in major markets like Western Europe and Japan [46][47] - The company has identified approximately 750 patients in the U.S. with RDEB, with a significant opportunity for repeat treatments [49][51] Competitive Landscape - ZivaSkin differentiates itself by providing long-lasting wound coverage with a single application, addressing both wound healing and quality of life improvements [53][54] Partner Programs - Abeona is eligible for royalties and milestones from partnered programs with Ultragenyx and Atacea, which are not factored into current projections [55][58] Key Milestones Ahead - The company aims to achieve its treatment goals for ZivaSkin and activate additional qualified treatment centers, with several PDUFA action dates for partnered programs expected in 2025 [66][67]

Pacira BioSciences Inc (PCRX) 2025 Conference June 04, 2025 08:45 AM ET Speaker0 Hi, good morning. Welcome to the Jefferies Healthcare Conference in New York, day one. My name is Dennis Singh, biotech analyst here at Jefferies. I have the pleasure of having Paseer Biosciences CEO, Frank Lee, here with us. Welcome. Frank, maybe to kind of kick things off, just give a brief overview of the company for those who aren't familiar. Just talk about the business and, you know, how things have evolved over the last ...

Recently disclosed clinical cohort data from high (1x1015 total vg) and low dose (5.7x1014 total vg) TSHA-102 from REVEAL adolescent/adult and pediatric Phase 1/2 trials Caregiver research regarding gain/regain of developmental milestones supporting alignment with FDA on primary endpoint in the pivotal Part B trial of TSHA-102 Previously disclosed preclinical data supporting broad biodistribution across brain and spinal cord regions following lumbar intrathecal delivery of AAV9 gene therapy vectors in non-h ...

Study met primary endpoint of ≥15-letter (≥3-line) gain in mesopic low contrast distance visual acuity in comparison to placeboPhase 3 study showed patient-reported functional benefit in treating significant, chronic night driving impairment in keratorefractive patients with reduced mesopic vision, a condition with no current FDA-approved therapiesSafety profile consistent with previous studies, with no new safety signals identified No evidence of tachyphylaxis was observed in this study over the 6-week per ...

Core Insights - uniQure N.V. is advancing its investigational gene therapy AMT-130 for Huntington's disease, with a Biologics License Application (BLA) submission planned for the first quarter of 2026, following alignment with the FDA on key components of the statistical analysis plan and Chemistry, Manufacturing and Controls (CMC) information [1][2][8] Regulatory Update - The FDA has supported the use of the composite Unified Huntington's Disease Rating Scale (cUHDRS) as an acceptable clinical endpoint for accelerated approval, with the primary efficacy analysis focusing on the 3-year change in cUHDRS in high-dose AMT-130 patients compared to an external control arm [3][4] - The ENROLL-HD dataset, which includes approximately 33,000 patients, will serve as the external control dataset for the primary analysis, enhancing the robustness of the statistical analysis plan due to its larger sample size and lower attrition rates [4][5] Chemistry, Manufacturing and Controls (CMC) - The FDA has agreed that the validation of the AMT-130 manufacturing process can leverage prior knowledge from the HEMGENIX® process, along with additional full-scale AMT-130 GMP batches and a single Process Performance Qualification (PPQ) batch [6][7] Next Steps - The company plans to submit an updated statistical analysis plan to the FDA in Q2 2025, initiate the PPQ run and present topline Phase I/II data in Q3 2025, hold a pre-BLA meeting in Q4 2025, and submit the BLA in Q1 2026 with a request for priority review designation [15] Clinical Program Overview - uniQure is conducting two multi-center, dose-escalating, Phase I/II clinical studies to evaluate the safety and efficacy of AMT-130, with a total of 26 patients in the U.S. study and 13 patients in the European study, exploring both low and high doses [10][11] - AMT-130 has received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation and Breakthrough Therapy designation, marking it as the first therapy for Huntington's disease to achieve RMAT designation [11] Huntington's Disease Context - Huntington's disease is a rare neurodegenerative disorder affecting approximately 70,000 diagnosed individuals in the U.S. and Europe, with no approved therapies currently available to slow its progression [12] Company Background - uniQure is focused on gene therapy, with a pipeline that includes treatments for Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease, building on its historic achievement in gene therapy for hemophilia B [13]