Core Insights - ZEVASKYN™ (prademagene zamikeracel) is the first FDA-approved autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][2][9] - The pivotal Phase 3 VIITAL study demonstrated significant wound healing and pain reduction after a single treatment, with 81% of treated wounds showing at least 50% healing compared to 16% in control wounds [5][13] - The publication of the VIITAL study results in The Lancet marks a significant milestone for Abeona Therapeutics as it prepares for the U.S. launch of ZEVASKYN [3][4] 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 fully integrated cGMP cell and gene therapy manufacturing facility in Cleveland, Ohio, which is responsible for the commercial production of ZEVASKYN [15] Study Details - The VIITAL study was an intra-patient randomized, open-label, controlled Phase 3 trial involving 11 patients with RDEB, assessing 43 pairs of large chronic wounds [3][4] - The study's co-primary endpoints of wound healing and pain reduction were successfully met, with significant statistical differences observed [5][13] Clinical Results - At week 24, 65% of treated wounds achieved 75% or more healing compared to 7% of control wounds, indicating a substantial improvement in treatment efficacy [13] - Mean change in wound pain was -3.1 in treated wounds versus -0.9 in control wounds, demonstrating a significant reduction in pain levels [13] Safety Profile - No serious ZEVASKYN-related adverse events were reported, consistent with previous clinical experiences, and no cases of squamous cell carcinoma were observed in treated wounds [13][14]

Addressing quality challenges is essential to ensure the safety and quality of biotherapeutics. Both USP and Bio-Techne have a long history of developing solutions to support mAbs and are applying their know-how to solving analytical challenges in AAV-based gene therapies. The ability to purchase USP reference standards from Bio-Techne further simplifies the method development process, enabling customers to assess system suitability and other critical criteria for charge and size based analytical assays on ...

Core Insights - Fractyl Health's Rejuva platform shows promise as a one-time, durable therapy for obesity and type 2 diabetes (T2D), demonstrating a favorable toxicity profile in preclinical studies [1][2][4] - The mechanism of action for Rejuva is distinct from systemic GLP-1 receptor agonists, allowing for nutrient-responsive secretion that activates only under metabolic stress [2][3] - Preliminary clinical data for RJVA-001, the first clinical candidate from the Rejuva platform, is expected in 2026, pending regulatory authorization [5][11] Key Findings - A single dose of Rejuva in a study with mice resulted in a 20% reduction in body weight and a 38% decrease in blood glucose levels by day 21, effects maintained despite continued high-fat diet exposure [7] - Rejuva-treated lean animals showed no weight gain or hyperglycemia when switched to a high-fat diet, maintaining baseline body weight and showing an 8% reduction in blood glucose [7] - The treatment was well-tolerated with no signs of toxicity or excessive weight loss in healthy animals, reinforcing the self-limiting nature of Rejuva's mechanism [7] Momentum Toward the Clinic - Fractyl Health has completed key in vivo studies for RJVA-001 and anticipates dosing the first patients in 2026, aiming for a first-in-class gene therapy that reprograms the pancreas for natural metabolic control [5][11] Scientific Validation - The study was conducted by a team from Fractyl Health and the University of Michigan, including experts in metabolic diseases, highlighting the scientific momentum behind Rejuva's approach [6][8]

PBFT02 continued to demonstrate robust, durable elevation in CSF PGRN levels and improvement in plasma NfL, a disease progression biomarker, compared to natural history Dose 2, 50% lower than Dose 1, substantially increased CSF PGRN levels at 30-days, reaching the upper limit of a healthy adult reference range Plan to amend upliFT-D protocol to include a prophylactic course of low dose anticoagulation and modify inclusion criteria to study patients earlier in disease progression Remain on track to seek regu ...

>> All right. Welcome back, everybody. The Trump administration has cut health research funding to $2.8% billion in May.That's down 28% from April. That's according to data from the Treasury Department. Joining us right now to discuss the implications is former FDA Commissioner Scott Gottlieb.He's 2828. It's 2.8%, not 28. He's a board member with Pfizer and Illumina.Also a CNBC contributor. Doctor Gottlieb, thanks for being here today. First of all, describe what happened.Why was the funding cut and where w ...

Core Viewpoint - Roche has implemented new dosing restrictions for Elevidys™ (delandistrogene moxeparvovec) for non-ambulatory Duchenne muscular dystrophy (DMD) patients due to two cases of fatal acute liver failure, leading to a reassessment of the benefit-risk profile as unfavorable for this patient group [1][2][4][7]. Group 1: Dosing Restrictions - Effective immediately, non-ambulatory patients will no longer receive Elevidys in commercial settings, and dosing for these patients in clinical trials is paused until additional risk mitigation measures are established [1][7]. - The new restrictions do not affect ambulatory DMD patients, where the benefit-risk ratio remains positive [2][7]. Group 2: Clinical Context - The two fatal cases of acute liver failure occurred among approximately 140 non-ambulatory patients treated with Elevidys globally [4]. - European regulators have requested temporary clinical holds on several Elevidys studies following the incidents [4]. Group 3: Product Overview - Elevidys is the first approved gene therapy for DMD, designed to deliver new instructions to cells to produce dystrophin, aiming to slow disease progression [10][11]. - The treatment has been approved in eight territories, including Bahrain, Brazil, and Japan, and Roche collaborates with Sarepta Therapeutics for its commercialization [5][11]. Group 4: Disease Background - Duchenne muscular dystrophy is a rare genetic disease primarily affecting males, with a prevalence of 1 in 5,000 boys born worldwide, leading to progressive muscle weakness and loss of mobility [3][12]. - The average life expectancy for individuals with Duchenne is only 28 years, highlighting the critical need for effective treatments [12][16].

Regeneron Pharmaceuticals (REGN) FY 2025 Annual General Meeting June 13, 2025 10:30 AM ET Speaker0 Good morning, and welcome to the twenty twenty five Annual Shareholder Meeting of Regeneron Pharmaceuticals. The meeting will begin after the following message. Speaker1 At Regeneron, we've always pursued bold science to drive our purpose, transforming lives through groundbreaking innovation. That principle is just as strong today as it was when the company was founded. Speaker2 We committed to not just treati ...

BURLINGTON, Mass., June 13, 2025 (GLOBE NEWSWIRE) -- Fractyl Health, Inc. (Nasdaq: GUTS) (the Company), a metabolic therapeutics company focused on pattern-breaking approaches to treat the root causes of obesity and type 2 diabetes (T2D), today announced it will present a poster of new preclinical data from its Rejuva Smart GLP-1 pancreatic gene therapy platform at the American Diabetes Association’s (ADA) 85th Scientific Sessions being held June 20-23, 2025, in Chicago, Illinois. Poster Presentation Detail ...

Pharvaris (PHVS) FY Conference June 11, 2025 04:00 PM ET Speaker0 All right. Good afternoon, and welcome to our, I think, the final session the conference. And it is my pleasure to introduce Maggie Beller, the head of IR for Verus. Maggie, welcome. I know this is the time I'm hosting you here. And before I go to the Q and A, let me just turn it to you for a quick introduction of the company, and maybe a brief overview about what's exciting ahead, and some of the catalysts and timing for that. Speaker1 Absol ...

Summary of 4D Molecular Therapeutics (FDMT) FY Conference Call Company Overview - **Company**: 4D Molecular Therapeutics (FDMT) - **Focus**: Gene therapy with a focus on ophthalmology and pulmonology, specifically targeting conditions like wet age-related macular degeneration (AMD), diabetic macular edema (DME), and cystic fibrosis. Key Points Industry and Market Opportunities - **Gene Therapy Potential**: The company believes it has developed best-in-class vectors for various delivery methods, which opens up large sustainable markets in gene therapy [1][2] - **Market Size**: The potential market opportunities are significant, with estimates of 4,150 million for wet AMD and DME, and 4,710 million for cystic fibrosis lung disease [1] Clinical Development and Trials - **Phase III Trials**: The focus for the next twelve months is on executing Phase III trials for FORWARD I and FORWARD II in wet AMD and initial dose data for cystic fibrosis [2] - **Physician Feedback**: Positive feedback from physicians regarding the durability of treatments and reduced injection burden, with expectations of an 80-90% reduction compared to existing therapies [6][7] - **Safety Profile**: The company reports a strong safety profile with no inflammation observed in DME trials, which is critical for large markets like wet AMD and DME [9][10] Commercial Strategy - **Pricing Flexibility**: The cost of goods for treatments is around $500, allowing for competitive pricing strategies that can benefit patients and shareholders [26] - **Durability Demand**: Surveys indicate that durability and extended treatment duration are the top desires for both patients and physicians, positioning gene therapy as a leading option [22] Regulatory and Financial Position - **Regulatory Engagement**: Ongoing discussions with the FDA regarding potential accelerated approval pathways for cystic fibrosis treatments, especially for patients with no available therapies [41][43] - **Financial Health**: The company ended Q1 with $458 million in cash, primarily allocated to fund Phase III trials for wet AMD, with a runway extending into 2028 [51] Future Directions - **Partnership Opportunities**: The company is open to partnerships outside the U.S. for its products, particularly in the lung franchise, while retaining U.S. rights for ophthalmology [48][49] - **Platform Development**: The gene therapy platform has shown broad utility, with six open INDs and a focus on developing a portfolio of products efficiently [45][54] Additional Insights - **Market Differentiation**: The company emphasizes that not all gene therapies are the same, highlighting its unique approach to local application and cost management, which differentiates it from other programs in the field [56] This summary encapsulates the key insights and strategic directions discussed during the conference call, providing a comprehensive overview of 4D Molecular Therapeutics' current status and future outlook in the gene therapy market.