Nanoscope Therapeutics has received Pioneering Regenerative Medical Product (Sakigake) and Orphan Drug designations for ...

Japan's Ministry of Health, Labour and Welfare (MHLW) grants MCO-010 Sakigake (Pioneering Regenerative Medical Product) and Orphan Drug designations, enabling an accelerated regulatory pathway for ...

Panelists discuss how emerging therapies—including TKIs, sustained-release implants, and gene therapy—may reduce treatment ...

Gene therapy emerges as a transformative force in ophthalmology, offering renewed hope for patients with retinal diseases. This innovative approach targets both inherited and acquired conditions, ...

Researchers have demonstrated the ability to deliver a fully functional copy of the CLN3 gene to stem cells of patients with juvenile NCL, an inherited neurodegenerative disease in which a mutation in ...

Two of Opus's seven gene therapy assets are in clinical trials, with additional programs expected to enter the clinic.

Opus Genetics, Inc., a clinical-stage biotechnology company focusing on gene therapies for inherited retinal diseases, announced upcoming presentations at various scientific conferences in May 2025.

We came across a bullish thesis on REGENXBIO Inc. on BioEquity Watch’s Substack. In this article, we will summarize the bulls ...

MALVERN, Pa., April 29, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (OCGN) (Ocugen or the Company), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the ...

Avista Therapeutics, a preclinical gene therapy company built on deep clinical, computational and gene therapy expertise, today announced the significant expansion of its gene therapy platform.

Researchers are expressing optimism that gene therapy can treat a wide range of eye diseases, including some that currently have no treatments. Only one gene therapy has been approved by the US Food ...