Gene-editing therapy proves effective for Leber's hereditary optic neuropathy

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

Ultragenyx Pharmaceutical (RARE) resubmits biologics license application for gene therapy treatment

Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) is one of the 13 Best Revenue Growth Stocks to Buy Right Now. On January 30, ...
NC Biotech

AskBio secures IND acceptance for late-onset Pompe disease gene therapy

AskBio Inc., an RTP-based gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has received U.S. Food and Drug Administration (FDA) acceptance of its ...

Bionano Announces Publication from Sanford Burnham Prebys Medical Discovery Institute Describing use of OGM to Detect Genomic Alterations Introduced by Gene Editing Technologies

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 ...

Affinia Therapeutics Announces FDA Acceptance of IND Application to Advance AFTX-201 to a Phase 1/2 Trial for the Treatment of BAG3-Associated Dilated Cardiomyopathy (DCM)

The UPBEAT (c) clinical trial is a multicenter, open-label Phase 1/2 clinical trial designed to evaluate the safety, tolerability, pharmacodynamics, and preliminary efficacy of AFTX-201 in adults with ...
Yicai Global

Chinese Scientists Make Key Advance in Using Gene Therapy

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

Rare stock slips 3% today — what’s up with Ultragenyx’s gene therapy for Sanfilippo syndrome?

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

Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA

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

Fractyl Health Submits Clinical Trial Application for First-in-Human Study of RJVA-001 Gene Therapy for Type 2 Diabetes and Obesity

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 ...
Fred Hutch

Scientists see foam as starting point of a path to bedside gene therapy

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...
PharmiWeb

Cell and Gene Therapy Market Report Predictions by Global Market Trends, Future Growth, Regional Overview and Forecast Outlook until 2030

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 ...
EurekAlert!

Breakthrough in Gene-Editing Therapy for Leber’s Hereditary Optic Neuropathy

A research team led by Professor Hyunji Lee of the Department of Convergence Medicine at Korea University College of Medicine (co-first authors: PhD candidates Sanghun Kim, Ji Eun Kim, and Sungjin Ju; ...