Lilly and German biotech Seamless Therapeutics will develop recombinase-based treatments for hearing loss.

Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...

Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotypeBLA resubmitted to U.S. FDA in January 2026; ...

The FDA in July last year declined to approve UX111 for Sanfilippo syndrome, a rare neurodegenerative disorder, citing ...

Apis Capital Advisors’ Apis Flagship Fund released its Q4 2025 investor letter. A copy of the letter can be downloaded here.

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...

With a shared purpose, we can realize the true promise of CRISPR and improve healthcare, providing hope to more patients with ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

The FDA AMT designation verifies NanoMosaic platform's ability to enhance efficiency, product quality, and scalability across AAV gene therapy manufacturing.

Gazi University in Ankara has officially launched the production process for Türkiye's first local and national gene therapy research product. The project, which targets rare genetic diseases, is ...