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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.

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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 325
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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 337
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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 357
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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 335
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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 327
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New gene therapy for muscular dystrophy offers hope

Science Daily: Pharmacology News

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

Therapies 302