Core Points - ZEVASKYN is the first and only cell-based gene therapy approved for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [2][8] - Ann & Robert H. Lurie Children's Hospital of Chicago is the first Qualified Treatment Center (QTC) for ZEVASKYN, with treatments expected to begin in Q3 2025 [1][3] - Abeona Therapeutics has launched the Abeona Assist™ program to provide personalized support for patients and families throughout the treatment journey [1][5] Company Overview - Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with ZEVASKYN being a key product in its portfolio [15] - The company operates a cGMP manufacturing facility in Cleveland, Ohio, which is responsible for the commercial production of ZEVASKYN [15] Treatment Details - ZEVASKYN incorporates the COL7A1 gene into a patient's skin cells to produce functional type VII collagen, which is essential for skin integrity [8] - The therapy has shown clinically meaningful results in wound healing and pain reduction with a single application [8] Hospital and Research Center - Lurie Children's Hospital has been a center of excellence for genetic skin diseases for over 30 years and is recognized for its expertise in treating epidermolysis bullosa [3][12] - The hospital has been providing FDA-approved gene therapies since 2019 and is expanding its offerings, with ZEVASKYN being the tenth gene therapy available [4]

Lineage Cell Therapeutics (LCTX) Q1 2025 Earnings Call May 13, 2025 04:30 PM ET Speaker0 Welcome to the Lineage Cell Therapeutics First Quarter twenty twenty five Conference Call. At this time, all participants are in a listen only mode. An audio webcast of this call is available on the Investors section of Lineage's website at www.lineagecell.com. This call is subject to copyright and is the property of Lineage, and recordings, reproductions, or transmissions of this call without the express written consen ...

Presenting new clinical data from Phase 1/2 multi-center clinical trial of LYL314, a next-generation dual‑targeting CD19/CD20 CAR T-cell product candidate for the treatment of relapsed and/or refractory large B‑cell lymphoma at the 18th International Conference on Malignant Lymphoma.Received Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA) for LYL314 for the treatment of relapsed and/or refractory diffuse large B-cell lymphoma in the third- ...

64% (9/14) complete response rate with AlloNK + rituximab in heavily pretreated patients that were naïve to prior CAR-T cell therapy, in line with approved auto-CAR-T therapies in aggressive B-NHL Median duration of response for AlloNK + rituximab not yet reached and is at least 19.4 months, in line with approved auto-CAR-T therapies in aggressive B-NHL AlloNK’s activity and well-tolerated safety profile in aggressive B-NHL patients support the potential to deliver deep B-cell depletion in a refractory auto ...

Enrollment complete in registrational Phase 2 KYV-101 trial in stiff person syndrome (SPS); on track for topline data in 1H 2026; biologics license application (BLA) filing now anticipated in 1H 2026 Advancing into registrational Phase 3 KYV-101 trial in myasthenia gravis (MG) following successful end-of-Phase 2 meeting with U.S. Food and Drug Administration (FDA); Phase 2 MG data anticipated in 2H 2025Company to host KOL event in Q3 2025, spotlighting its accelerating neuroinflammation franchise Strong fin ...

Summary of Sana Biotechnology Conference Call Company Overview - **Company**: Sana Biotechnology (SANA) - **Event**: 2025 Bank of America Healthcare Conference - **Date**: May 13, 2025 Key Points on Type One Diabetes - **Disease Impact**: Type one diabetes affects approximately nine million people globally, leading to a reduced lifespan of 10-15 years and significant daily challenges for patients [4][5] - **Unmet Medical Need**: No new drug has been developed for type one diabetes since insulin in 1923, indicating a substantial unmet need for better treatment options [5] - **Current Research**: Sana is focusing on gene-modified pluripotent stem cells to create a scalable source of pancreatic islets for transplantation, aiming to eliminate the need for immunosuppression [10][12] Recent Data and Developments - **Clinical Data**: A patient treated with gene-modified islet cells has been insulin-free for over 30 years, with no signs of immunologic response after 12 weeks [10][12] - **Future Plans**: An Investigational New Drug (IND) application is anticipated next year, with the goal of beginning human treatments [11][12] Challenges and Considerations - **Scaling Production**: Significant work is required to scale the production of gene-modified islets to meet the needs of the nine million affected individuals [18][20] - **Manufacturing Process**: The manufacturing process must be locked in before starting pivotal studies, which is expected to take about a year [29] Other Pipeline Assets - **CAR T Cell Therapy**: Sana is applying hypoimmune technology to develop allogeneic CAR T cells, addressing challenges in the autoimmune setting and aiming for improved patient convenience [31][32] - **Ongoing Studies**: Two studies, Gleam and Vivid, are ongoing, with data expected later this year [30] Market and Financial Considerations - **Investment Landscape**: The company acknowledges the need for increased investment to continue development, particularly in the competitive field of cell and gene therapy [39][40] - **Return on Investment**: The potential for high ROI is emphasized, contingent on successful scaling and safety of the diabetes treatment [43][44] Regulatory Environment - **FDA Interactions**: The company is optimistic about upcoming meetings with the FDA to confirm testing protocols for their gene-modified therapies [17] Conclusion - **Commitment to Diabetes Asset**: Sana Biotechnology is committed to advancing its diabetes treatment while navigating the complexities of scaling production and ensuring safety [42]

Legend Biotech (LEGN) Q1 2025 Earnings Call May 13, 2025 08:00 AM ET Company Participants Caroline Paul - Associate Director - IRYing Huang - CEOAlan Bash - President - CARVYKTIJessie Yeung - Interim CFOGena Huidong Wang - Managing Director, Biotech Equity ResearchGuowei Fang - President - Research & DevelopmentMythili Koneru - Chief Medical OfficerClara Dong - Vice President - Biotechnology Equity ResearchJonathan Miller - Managing DirectorJames Shin - Director of Biopharma Equity ResearchAshwani Verma - E ...

Financial Data and Key Metrics Changes - The company reported net sales of $4 million for Q1 2025, with a revenue guidance for the full year of $35 million to $45 million from TESELRA sales [3][4][5] - The average turnaround time from apheresis to release was 27 days, beating the target of 30 days [5] - The company achieved a 100% manufacturing success rate from its U.S. T Cellular manufacturing center [5][6] Business Line Data and Key Metrics Changes - A total of 21 patients have been treated with TESELRA in 2025, with 13 in Q1 and 8 in early Q2 [3][4] - The company invoiced 14 T Cellular treatments in 2025 to date, with 6 in Q1 [4] - The company anticipates peak sales of $400 million from its combined T Cellra and Letocell sarcoma franchise [5] Market Data and Key Metrics Changes - The company has established 28 authorized treatment centers (ATCs) for TESELRA, with plans to reach approximately 30 by the end of 2025 [3][4] - The company expects to treat around 1,000 patients annually diagnosed with synovial sarcoma, aligning with initial forecasts [67][68] Company Strategy and Development Direction - The company is focused on the successful launch of TESELRA and preparing for the launch of Letocell, anticipated in 2026 [6] - Investments in infrastructure for T Cellra will also support the Letocell launch [6] - The company is exploring strategic options with Cowen as advisors to benefit patients and shareholders [7] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving the revenue guidance based on patient treatment cycles and the number of treatment centers [3][5] - The management noted that the FDA is engaged and working diligently on regulatory matters, with no indications of delays [28] - The company has seen positive trends in patient referrals and screening, expecting incremental growth quarter over quarter [18][19] Other Important Information - The company has not experienced any patient denials to date, indicating effective patient access to TESELRA [5] - The company is managing its balance sheet and has paid down $25 million of financing obligations to improve leverage [44][45] Q&A Session Summary Question: Clarification on apheresis patients invoiced - Management indicated that most apheresis patients invoiced in Q1 were from the prior quarter, with expectations for invoicing to continue in the coming months [10] Question: Trends in patient referrals and screening - Management expects incremental growth quarter over quarter, driven by increased awareness and onboarding of ATCs [18] Question: Impact of regulatory changes - Management noted ongoing engagement with the FDA and no indications of delays in regulatory processes [28] Question: Key learnings from the early launch - Management highlighted faster-than-expected onboarding of treatment centers and a 100% manufacturing success rate as key positives [30][31] Question: Cash runway and sustainability of operations - Management acknowledged a substantial debt concern and indicated that cash runway guidance is inappropriate due to various impacting factors [45][46] Question: Drop-off rate from apheresis to infusion - Management confirmed that there have been no cancellations from the moment a patient is enrolled, indicating a high conversion rate [39] Question: Manufacturing maintenance plans - Management stated there are no significant maintenance plans that would impact capacity for the year [58] Question: COGS tracking and expectations - Management indicated that COGS for the first few quarters will be higher than expected due to pre-purchased products, with margins expected to normalize [63][66]

Nektar Therapeutics (NKTR) Q1 2025 Earnings Call May 08, 2025 05:00 PM ET Company Participants Corinne Franklin - Investor RelationsHoward Robin - President and Chief Executive OfficerJonathan Zalevsky - Chief Research & Development OfficerSandra Gardiner - Chief Financial OfficerJulian Harrison - Managing DirectorMayank Mamtani - Senior Managing DirectorYu He - VP - Equity Research Conference Call Participants Yasmeen Rahimi - Sr. Research AnalystJay Olson - Managing Director & Senior Analyst - Biotechnolo ...

Nektar Therapeutics (NKTR) Q1 2025 Earnings Call May 08, 2025 05:00 PM ET Speaker0 Good day, and thank you for standing by. Welcome to the Nectar Therapeutics First Quarter twenty twenty five Financial Results Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer session. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker today, Corinne Franklin in ...