Biotech innovation stories

Sep 21, 2026

Beacon Therapeutics' gene therapy succeeds in phase 2 trial for rare inherited eye disease

Beacon Therapeutics reported its experimental gene therapy improved low-light reading ability in a late-stage trial for X-linked retinitis pigmentosa (XLRP), a rare inherited eye disease that can cause blindness. The company succeeded where larger drugmakers including Johnson & Johnson and Biogen have failed.

Sep 21, 20262 sources

Sep 14, 2026

Scholar Rock's spinal muscular atrophy drug Isembyld approved by FDA

The FDA approved Isembyld (apitegromab), the first muscle-targeted treatment for spinal muscular atrophy. The drug will cost approximately $310,000 annually and showed improvements in motor function in clinical trials.

Sep 14, 20263 sources

Aug 26, 2026

RevMed's pancreatic cancer pill daraxonrasib wins FDA approval at $475,000 annual cost

Revolution Medicines' breakthrough pill targeting KRAS mutations in advanced pancreatic cancer received FDA approval, priced at over $475,000 per year. The drug nearly doubles survival in patients with this hard-to-treat cancer.

Aug 26, 20262 sources

Aug 20, 2026

Ultragenyx wins first gene therapy approval with Genglycos for glycogen storage disease

The FDA approved Ultragenyx's Genglycos gene therapy for a rare glycogen storage disease, marking the company's first gene therapy clearance and providing a priority review voucher.

Aug 20, 20264 sources

Jun 30, 2026

FDA Approves Orca Bio's TREGZI as First Precision-Engineered Cell Therapy for Blood Cancer Transplants

The U.S. FDA has approved Orca Bio's TREGZI, the first precision-engineered cell therapy for allogeneic transplants in adults with hematological malignancies, designed to reduce stem cell transplant complications.

Jun 30, 20267 sources

Top claims

▪Beacon Therapeutics Chief Executive Officer Lance Baldo stated that the company has a chronological advantage as the only company with a positive readout on its primary endpoint within a pivotal phase three study for X-linked retinitis pigmentosa
▪Beacon Therapeutics announced plans to hold discussions with global regulatory authorities regarding a marketing application submission based on the results of its 12-month trial of 85 male subjects with X-linked retinitis pigmentosa treated with its gene therapy, laru-zova
▪According to National Institutes of Health data, X-linked retinitis pigmentosa affects approximately 4 in 100,000 males across the United States and Europe, and currently has no approved treatments

Subtopics

Drug Approval and Clinical Trials5 FDA drug approval4 Clinical trials2 Gene therapy2 Cancer treatment1 Cancer treatment breakthroughs1 Drug pricing1 Precision medicine1

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