OGM was selected as a genome-wide, unbiased method to detect large genomic rearrangements and structural variants (SVs) with sensitivity to variant allele fractions (VAFs) as low as 5% to analyze ...

Taysha Gene Therapies recently advanced its TSHA-102 Rett syndrome program, dosing the first patient in the REVEAL pivotal phase and receiving FDA authorization to use ASPIRE trial data in a planned ...

Ultragenyx now expects a 6-month review period for the therapy and a likely decision from the FDA regarding potential ...

(Yicai) Jan. 28 -- Chinese medical researchers have made a breakthrough in gene therapy that overcomes the limitations of ...

Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical ...

Affinia Therapeutics ("Affinia"), an innovative gene therapy company with a pipeline of first-in-class and/or best-in-class ...

Researchers developed an AAV platform that reconstitutes large genes in vivo, achieving robust expression and therapeutic benefits. This strategy offers therapeutic possibilities for previously ...

Fractyl Health announced a first-in-human trial for RJVA-001, targeting T2D and obesity, pending regulatory approval by 2026. Fractyl Health, Inc. has announced the submission of the first module of ...

A revolutionary treatment brings new hope to those suffering from a devastating rare skin disorder, while showcasing an innovative funding model that could transform rare disease research In a ...

A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...

Market OverviewThe global cell and gene therapy market is undergoing rapid and transformative growth, reflecting its increasing importance in modern healthcare.The market is projected to expand at a ...

Sanofi’s participation will support the progression of Sensorion’s genetic medicine pipeline.