article thumbnail

Novel stem cell therapy repairs 'irreversible' corneal damage in clinical trial

Science Daily: Pharmacology News

Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.

article thumbnail

100x improvement in sight seen after gene therapy trial

Science Daily: Pharmacology News

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

Therapies 323
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Duchenne approval exposes FDA rift over Sarepta gene therapy

BioPharma Drive: Drug Pricing

Peter Marks’ decision to override the objections of agency staff and broaden use of Elevidys could have a “lasting impact” on gene therapy as well as the FDA, one analyst wrote.

Therapies 334
article thumbnail

Cell and gene therapy investment, once booming, is now in a slump

BioPharma Drive: Drug Pricing

Far fewer venture funding rounds were closed by cell and gene therapy developers over the first six months of 2024 than in prior years. Experts say there are several factors.

Therapies 356
article thumbnail

FDA endorses speedy approval path for Regenxbio Duchenne gene therapy

BioPharma Drive: Drug Pricing

The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne gene therapies despite questions about their effectiveness.

Therapies 332
article thumbnail

Artiva prices $167M IPO, riding optimism for autoimmune cell therapy

BioPharma Drive: Drug Pricing

The offering comes days after the publication of a paper showing what analysts said was early proof that “off-the-shelf” cell therapies can treat inflammatory diseases.

Therapies 324
article thumbnail

Early Hints at a Gene Therapy Cure for Herpes

Drugs.com

WEDNESDAY, May 15, 2024 -- An experimental gene therapy could one day provide a first-ever cure for genital and oral herpes, researchers report.The gene therapy removed 90% or more of oral herpes infection in lab mice, and it also suppressed how.

Therapies 298