MeiraGTx reacquired bota-vec, a treatment for X-linked retinitis pigmentosa (XLRP), from Johnson & Johnson for a $25 million upfront payment. The company raised $100 million through a share offering and plans to expedite regulatory filings for U.S. and EU approval with a potential 2027 launch. Three-year data from its xerostomia treatment showed durable benefits. Stock declined 15.8% on the announcement.

MeiraGTx has entered into an asset purchase agreement with Johnson & Johnson to acquire all interests in botaretigene sparoparvovec (bota-vec) for treating X-linked retinitis pigmentosa. The company will pay $25 million upfront plus regulatory and commercial milestones, with royalties on global net sales. Phase 3 LUMEOS study data showed clinically meaningful improvements across multiple vision domains, with 45% of treated patients gaining >10 letters in low luminance visual acuity. MeiraGTx intends to file for regulatory approval in the U.S., EU, and Japan with a potential launch in 2027.

Eli Lilly signed a strategic collaboration with MeiraGTx for ophthalmology gene therapy, granting worldwide exclusive rights to AAV-AIPL1 program for Leber congenital amaurosis 4, with an upfront payment of $75 million and potential milestone payments over $400 million.

MeiraGTx reported Q2 2025 results with significant revenue growth but missed analyst estimates. The company advanced several gene therapy programs, secured key partnerships, and maintained strong manufacturing capabilities despite ongoing financial challenges.

A comprehensive analysis reveals over 70 companies developing AAV gene therapies for hereditary retinal diseases, offering potential transformative treatments for vision restoration through advanced gene therapy techniques.

Coave Therapeutics, a company pioneering genetic medicines, has established a new Scientific Advisory Board (SAB) with renowned experts in AAV vector biology, gene therapy for ophthalmology and CNS diseases, and extra-hepatic gene delivery. The SAB will provide strategic guidance to support Coave's R&D initiatives and the development of its ALIGATER platform.

The Parkinson's disease market is expected to grow significantly due to the introduction of emerging therapies, advancements in treatment options, and a rising prevalence of the condition.

The report provides a comprehensive analysis of the Adeno-Associated Virus (AAV) Vectors in Gene Therapy pipeline, highlighting the potential of this technology to revolutionize the treatment of genetic disorders. It examines the challenges in scalable production and safety, as well as the industry's focus on optimizing vectors for improved safety, specificity, and efficiency.

CRISPR Therapeutics (CRSP) delivered earnings and revenue surprises of 12.27% and 93.94%, respectively, for the quarter ended March 2024. Do the numbers hold clues to what lies ahead for the stock?

LONDON and NEW YORK, April 24, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (NASDAQ: MGTX), a vertically integrated, clinical stage gene therapy company, today announced that Alexandria Forbes, Ph.D., President and Chief Executive Officer, will participate in the following investor conferences:

LONDON and NEW YORK, April 18, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced the Company gave an oral presentation at the American Academy of Oral Medicine Annual Conference, being held from April 16-20, 2024, at the Hyatt Regency Grand Cypress in Orlando, FL.

MeiraGTx Holdings PLC (MGTX) delivered earnings and revenue surprises of -2.17% and 79.86%, respectively, for the quarter ended December 2023. Do the numbers hold clues to what lies ahead for the stock?