RESEARCH TRIANGLE PARK, N.C., May 12, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and other treatments for ophthalmic disorders, today announced presentations related to its IRD gene therapy programs at these upcoming scientific conferences in May. Presentation Details American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting Title:Evaluation of MERTK gene therapy ...

CANbridge Pharmaceuticals Corporate Presentation Aug 2023 Disclaimer THIS DOCUMENT OR THE INFORMATION CONTAINED HEREIN IS NOT INTENDED TO AND DOES NOT CONSTITUTE ANY OFFER OR INVITATION, SOLICITATION, COMMITMENT OR ADV ERTISEMENT OF ANY OFFER FOR SUBSCRIPTION, PURHCASE OR SALE OF ANY SECURITIES, NOR SHALL ANY PART OF THIS DOCUMENT FORM THE BASIS OF OR BE RELIED ON IN CONNECTION WITH ANY CONTRACT OR COMMITMENT WHATSOEVER. This document contains strictly conf idential and proprietary inf ormation in relation ...

Financial Performance - Rocket Pharmaceuticals incurred a loss of 56 cents per share in Q1 2025, which is narrower than the Zacks Consensus Estimate of a loss of 59 cents and an improvement from a loss of 66 cents per share in the same quarter last year [1] - The company did not record any revenues in the reported quarter, missing the Zacks Consensus Estimate for total revenues of $8 million [2] - General and administrative expenses rose 28% year over year to $28.4 million, attributed to increased commercial preparation and legal expenses [2] - Research and development expenses were $35.9 million, down 21% from the previous year due to reduced manufacturing and development costs [3] - As of March 31, 2025, the company had cash, cash equivalents, and investments totaling $318.2 million, down from $372.3 million as of December 31, 2024, with expectations to fund operations into Q4 2026 [3] - Year to date, RCKT shares have declined 44%, compared to an 8% decline in the industry [4] Pipeline Developments - Kresladi, developed for treating severe leukocyte adhesion deficiency-I (LAD-I), received a complete response letter (CRL) from the FDA in June 2024, requesting limited additional information on the Chemistry Manufacturing and Controls (CMC) [7][8] - The company plans to file a complete BLA to resolve the CRL later in 2025 [8] - Rocket Pharmaceuticals is developing RP-L102 for treating Fanconi anemia (FA) and has initiated a rolling BLA, expecting to complete the submission in late 2025 or early 2026 [9] - Dosing is currently underway in a phase II pivotal study for RP-A501, targeting male patients with Danon disease, with clinical data readout expected in mid-2026 [10]

Ocugen (OCGN) Q1 2025 Earnings Call May 09, 2025 08:30 AM ET Company Participants Tiffany Hamilton - AVP & Head of Corporate CommunicationsShankar Musunuri - Co-Founder, CEO & ChairmanRamesh Ramachandran - Chief Accounting OfficerHuma Qamar - Chief Medical OfficerArun Upadhyay - Chief Scientific Officer and Head of Research & Development Conference Call Participants Michael Okunewitch - Senior Biotechnology AnalystRobert Leboyer - Senior Biotechnology AnalystSwayampakula Ramakanth - Managing Director & Seni ...

Ocugen (OCGN) Q1 2025 Earnings Call May 09, 2025 08:30 AM ET Company Participants Tiffany Hamilton - AVP & Head of Corporate CommunicationsShankar Musunuri - Co-Founder, CEO & ChairmanRamesh Ramachandran - Chief Accounting OfficerHuma Qamar - Chief Medical OfficerArun Upadhyay - Chief Scientific Officer and Head of Research & Development Conference Call Participants Michael Okunewitch - Senior Biotechnology AnalystRobert Leboyer - Senior Biotechnology AnalystSwayampakula Ramakanth - Managing Director & Seni ...

uniQure (QURE) Q1 2025 Earnings Call May 09, 2025 08:30 AM ET Speaker0 Good day and thank you for standing by. Welcome to the UniCure First Quarter twenty twenty five Earnings 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. To ask a question during the session, you'll need to press 11 on your telephone. You will then hear an automated message advising your hand is raised. To answer your question, press ...

Ocugen (OCGN) Q1 2025 Earnings Call May 09, 2025 08:30 AM ET Speaker0 Good morning, and welcome to Ocugen's First Quarter twenty twenty five Financial Results and Business Update. Please note that this call is being recorded at this time. All participant lines are in listen only mode. Following the speakers' commentary, there will be a question and answer session. I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications. You may begin. Speaker1 Thank you, operator, and go ...

Conference Call and Webcast Today at 8:30 a.m. ET OCU400 Phase 3 clinical trial for retinitis pigmentosa (RP) is progressing well and on target for potential BLA/MAA filings by mid-2026Anticipate initiating OCU410ST Phase 2/3 pivotal confirmatory clinical trial for Stargardt disease mid- 2025 MALVERN, Pa., May 09, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today reported first quarter 2025 financia ...

~ AMT-130 granted Breakthrough Therapy designation by FDA ~ ~ Initial safety data from third cohort of Phase I/II study show AMT-130 continues to be generally well-tolerated, with no treatment-related serious adverse events ~ ~ Held Type B FDA meetings in the first and second quarters of 2025 to advance BLA preparations for AMT-130; regulatory update expected in the second quarter of 2025 ~ ~ AMT-260 clinical data from first patient to be presented at Epilepsy Therapies & Diagnostics Development Symposium o ...

OXB to Present at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) Oxford, UK – 9 May 2025: OXB (LSE: OXB), a global quality and innovation-led cell and gene therapy CDMO, today announces its participation in the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), taking place from 13–17 May 2025 at the Ernest N. Morial Convention Center in New Orleans, Louisiana. The Company will exhibit at Booth #1611, where delegates can meet OXB’s team of viral v ...