- Duchenne (SGT-003): Participant dosing ongoing in the Phase 1/2 INSPIRE DUCHENNE trial; Solid on track to discuss accelerated pathways with U.S. FDA later in 2025 - - FA (SGT-212): Dosing of first participant anticipated in the second half of 2025 - - CPVT (SGT-501): FDA IND on track for submission first half of 2025 - - Cash: Company ended first quarter 2025 with $306.9 million in cash, cash equivalents, investments and available-for-sale securities; Solid has anticipated cash runway into the first half ...

Taysha Gene Therapies Inc (TSHA) Q1 2025 Earnings Call May 15, 2025 08:30 AM ET Company Participants Hayleigh Collins - Senior Director - Corporate Communications & Investor RelationsSean Nolan - CEO & ChairmanSukumar Nagendran - President & Head - Research & DevelopmentKamran Alam - Chief Financial OfficerWhitney Ijem - Managing DirectorMalcolm Hoffman - Senior BioPharma Equity Research Associate Conference Call Participants Kristen Kluska - AnalystLydia Erdman - Biotech Equity Research AnalystGil Blum - S ...

Taysha Gene Therapies Inc (TSHA) Q1 2025 Earnings Call May 15, 2025 08:30 AM ET Speaker0 Greetings, and welcome to the Tatia Gene Therapies First Quarter twenty twenty five Earnings Call. At this time, all participants are in listen only mode. A question and answer session will follow the formal presentation. As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, Hayley Collins, Director, Head of Corporate Communications. Please go ahead. Speaker1 Thank ...

High dose and low dose of TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in the pediatric, adolescent and adult patients treated (high dose, n=6; low dose, n=4) across both REVEAL trials as of April 10, 2025, data cutoff Program update on pivotal trial design, Rett syndrome natural history data analysis and clinical data from cohort two (high dose) and cohort one (low dose) of both REVEAL trials expected in Q2 2025 in conjunction with IRSF Rett Syndrome Scientific Me ...

ARVO presentation highlights 12-month results from Phase 1/2 study that support potential of OPGx-LCA5 gene therapy to restore meaningful vision Pediatric cohort of LCA5 study ongoing with initial multi-patient data anticipated in Q3 2025 OPGx-BEST1 on track for IND filing and initiation of Phase 1/2 trial with early data expected in Q1 2026 Leading healthcare investors provide funding to deliver on key milestones RESEARCH TRIANGLE PARK, N.C., May 15, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IR ...

No serious treatment-emergent AEs related to the treatment or procedure were reported regardless of steroid pretreatment or dose level administered. Treatment-related joint effusions (swelling) were the most common AE, occurring in 36% of patients who received steroid pretreatment vs 61% of patients who were not pretreated. The majority of effusions were mild to moderate in severity and resolved in a median of 33 days among patients in the pretreated group. -- Early clinical data indicates that Pre-existing ...

Core Insights - Abeona Therapeutics has received FDA approval for ZEVASKYN™, the first autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][5] - The company is transitioning to a commercial-stage entity with the launch of ZEVASKYN and has activated its first treatment center [2][4] Recent Developments - ZEVASKYN's FDA approval marks a significant milestone for RDEB patients, with the first treatment expected in Q3 2025 [5] - Abeona has secured a $155 million non-dilutive capital through the sale of its Rare Pediatric Disease Priority Review Voucher (PRV), which will fund operations for over two years [3] - The company has entered into value-based agreements with commercial payer groups, ensuring access to ZEVASKYN for approximately 100 million commercially-insured lives [5] Financial Results - As of March 31, 2025, the company reported cash and cash equivalents totaling $84.5 million, down from $98.1 million at the end of 2024 [6] - Research and development expenses increased to $9.9 million in Q1 2025 from $7.2 million in Q1 2024, primarily due to scaling up manufacturing for ZEVASKYN [7] - The net loss for Q1 2025 was $12.0 million, a significant reduction from a net loss of $31.6 million in Q1 2024 [8][14]

LEXINGTON, Mass., May 15, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today announced the first peer-reviewed publication of data demonstrating the ability of alkaline phosphatase (ALPL) to transport a novel AAV capsid across the blood-brain barrier (BBB). The article, titled "Highly conserved brain vascular receptor ALPL mediates transport of engineered AAV vectors across the blood-brain barrier ...

Summary of 4D Molecular Therapeutics (FDMT) Conference Call Company Overview - **Company**: 4D Molecular Therapeutics (FDMT) - **Lead Product**: 4150, currently in phase three development for wet age-related macular degeneration (AMD) and diabetic macular edema (DME) [2][3] Industry Context - **Market Size**: The anti-VEGF market is valued at $17 billion and is expected to grow significantly due to an aging population [4] - **Competitive Landscape**: The market is competitive with existing therapies like aflibercept and new entrants like Babizmo, which has shown rapid uptake due to improved durability [4][5] Core Product Insights - **Durability**: 4150 aims to provide multi-year durability compared to existing therapies that offer only a few months of effectiveness [5][6] - **Injection Burden**: Current therapies require 8-10 injections per year, while 4150 could reduce this burden by 83-94% in various patient populations [7][8] - **Safety Profile**: The product has shown a favorable safety profile with no serious adverse events reported, and a low rate of mild transient effects [8][11] Clinical Development - **Phase Three Trials**: Top-line data for wet AMD pivotal trials is expected in the second half of 2027, designed to demonstrate non-inferiority in best corrected visual acuity (BCVA) and significant reduction in injection burden [12][20] - **Regulatory Designation**: 4150 has received RMAT designation for both wet AMD and DME, indicating a favorable regulatory outlook [3][16] Manufacturing and Commercialization - **Manufacturing Strategy**: Currently in-house manufacturing with plans to outsource as commercialization approaches, minimizing exposure to potential tariffs [19] - **Cost of Goods**: Estimated at $500, providing pricing flexibility and potential for favorable payer negotiations [11] Market Expectations and Physician Insights - **Initial Uptake**: Anticipated initial use in patients with high treatment burdens (8-10 injections/year), with gradual adoption in less severe cases as physician familiarity increases [29][30] - **Physician Feedback**: Physicians prioritize significant reductions in injection frequency over complete injection freedom, viewing an 80-90% reduction as a substantial benefit [22][24] Competitive Positioning - **Differentiation**: 4150 aims to set a new standard in durability, potentially offering years of effectiveness compared to competitors that may only extend treatment intervals by a few months [27][28] Additional Programs - **Other Developments**: Active program for 4710 targeting cystic fibrosis lung disease, with promising clinical activity expected to be updated later this year [13] Conclusion - 4D Molecular Therapeutics is positioned to disrupt the retinal vascular disease market with its innovative gene therapy, 4150, which promises significant improvements in treatment durability and patient outcomes while maintaining a strong safety profile. The company is on track for pivotal trial data and is strategically planning for commercialization amidst a competitive landscape.

BioCardia (BCDA) Q1 2025 Earnings Call May 14, 2025 04:30 PM ET Company Participants Miranda Peto Benvenuti - Investor RelationsPeter Altman - CEO, President & DirectorDavid McClung - Chief Financial OfficerLaura Suriel - Equity Research Associate Conference Call Participants Joseph Pantginis - MD & Senior Healthcare AnalystNone - Analyst Operator Participants of this call are advised that the audio of this conference call is being broadcast live over the Internet and is also being recorded for playback pur ...