Sep. 25, 2024 — Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an ...

The product candidate is a modifier gene therapy for broad retinitis pigmentosa indication.

The Asia-Pacific gene therapy market was valued at $349.1 million in 2020 and is expected to grow at a compound annual growth rate (CAGR) of 36.8% to reach nearly $7 billion in 2030. This ...

Children's Hospital of Michigan announced Monday that it now is treating the first person in Michigan — a teenage boy — with a newly approved gene therapy for the rare inherited blood disorder ...

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 ...

FRIDAY, Sept. 6, 2024 (HealthDay News) -- Gene therapy may restore vision to children and adults robbed of their sight by a rare inherited condition called Leber congenital amaurosis, researchers ...

This story first appeared in Adam’s Biotech Scorecard, a subscriber-only newsletter. STAT+ subscribers can sign up here to get it delivered to their inbox. The deep financial restructuring ...

Summary: A newly developed gene therapy shows great potential for treating glaucoma, a condition that can lead to vision loss. The therapy protects retinal ganglion cells, essential for vision, and ...

BridgeBio Pharma is slashing its gene therapy budget and pulling back from the modality after seeing the results of a phase 1/2 clinical trial. CEO Neil Kumar, Ph.D., said the data “are not yet ...

The startup’s technology combines an AAV — a common gene therapy delivery tool — with an immunotherapy approach now widely used in oncology. The idea is to give patients a one-time treatment that ...

"The era of gene therapy for neuromuscular diseases has begun, and the complexity of these diseases requires us to constantly innovate to improve drug candidates targeting muscle. The new ...

In 2019, around 1.5 million people in the U.S. were living with wet AMD. The potential reach of a gene therapy in this common disease space is in stark contrast to the current approved gene therapies ...