Genetics & Stem Cells News

Medindia's Press Release’ section provides the latest press release on Genetics & Stem Cells from across the world for the global audience. This page links to 1986 Genetics & Stem Cells press releases.

GenEditBio Announces First Patient Dosed in Investigator-Initiated Trial of GEB-101, World's First In Vivo CRISPR-Cas Ribonucleoprotein-Based Genome Editing Investigational Therapy for TGFBI Corneal Dystrophy

PR Newswire HONG KONG, BEIJING and BOSTON, June 6, 2025 HONG KONG, BEIJING and BOSTON, June 6, 2025 /PRNewswire/ -- GenEditBio Limited ("GenEditBio"), a clinical-stage biotechnology start-up company focusing on genome editing therapeutic ...


WuXi Biologics Congratulates Partner CANbridge Pharmaceuticals on the Approval of Innovative Velaglucerase-beta for Injection (Gaurunning) for Gaucher Disease by China NMPA

PR Newswire WUXI, China and SHANGHAI, May 16, 2025 WUXI, China and SHANGHAI, May 16, 2025 /PRNewswire/ -- WuXi Biologics (2269.HK), a leading global Contract Research, Development and Manufacturing Organization (CRDMO), congratulates its ...

Neurocrine Biosciences Presents Data on Improvements in Physiologic Glucocorticoid Dosing and Select Reproductive Hormones in Patients with Classic Congenital Adrenal Hyperplasia Taking CRENESSITY™ (crinecerfont)

PR Newswire SAN DIEGO, May 15, 2025 SAN DIEGO, May 15, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced new data from the Phase 3 CAHtalyst™ Adult and Pediatric studies of CRENESSITY™ (crinecerfont). The data ...

World's First Patient Treated with Personalized CRISPR Gene Editing Therapy at Children's Hospital of Philadelphia

PR Newswire PHILADELPHIA and NEW ORLEANS, May 15, 2025 Landmark Study from CHOP and Penn Medicine Showcases the Power of Customized Gene Editing Therapy to Treat Patient with Rare Metabolic Disease PHILADELPHIA and NEW ORLEANS, May 15, ...

Kaerus Bioscience's lead candidate KER-0193 granted Orphan Drug Designation and Rare Pediatric Drug Designation by U.S. FDA for treatment of Fragile X syndrome (FXS)

PR Newswire LONDON, May 14, 2025 LONDON, May 14, 2025 /PRNewswire/ -- Kaerus Bioscience ("Kaerus" or "the Company"), a clinical stage biopharmaceutical company created by Medicxi for the development of therapeutics for rare genetic syndromes ...

L2 Bio, FDA Approves Crohn's Disease Trial as a Phase 2b Utilizing Adipose-Derived Stem Cells

PR Newswire HENDERSON, Nev., May 13, 2025 FDA has approved an aggressive stem cell clinical trial, offering potential new relief through intravenous (IV) administration. HENDERSON, Nev., May 13, 2025 /PRNewswire-PRWeb/ -- "We at L2 Bio ...

MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome

PR Newswire MIDDLETON, Mass., May 12, 2025 First Patient Dosing Anticipated in Second Half of 2025  Additional Details to be Provided by Webinar in Coming Months  MIDDLETON, Mass., May 12, 2025 /PRNewswire/ -- MavriX Bio, a ...

Andelyn Biosciences Applies the AAV Curator® Platform to Deliver Novel Gene Therapy for an Ultra-Rare NEDAMSS Disease Patient 14-Months after Diagnosis

PR Newswire ?COLUMBUS, Ohio, May 12, 2025 ?COLUMBUS, Ohio, May 12, 2025 /PRNewswire/ -- Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has ...

Skyline Therapeutics Presents Late-Breaking Abstract on SKG1108, a Novel Optogenetic Gene Therapy for Retinitis Pigmentosa, at ASGCT 2025

PR Newswire SHANGHAI, May 10, 2025 SHANGHAI, May 10, 2025 /PRNewswire/ -- Skyline Therapeutics, an innovation-driven clinical-stage gene therapy company focused on developing unique and novel solutions for rare and severe diseases, today ...

Genprex Signs Exclusive License to Additional Gene Therapy Technology with UTHealth Houston for the Treatment of Glioblastoma

PR Newswire AUSTIN, Texas, May 6, 2025 License Expands Genprex's Portfolio of Oncology Gene Therapy Technologies AUSTIN, Texas, May 6, 2025 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene ...

Genetics & Stem Cells News »

 A Genetic Storybook: Mutation Sparks Igniting the Story of Life

A Genetic Storybook: Mutation Sparks Igniting the Story of Life

New study finds genetic modifiers control human mutation rates, revealing how populations evolve and risk for genetic diseases may vary.

Fanconi Anemia: Gene Discovery Opens New Doors for Treatment

Fanconi Anemia: Gene Discovery Opens New Doors for Treatment

New gene discovery in Fanconi Anemia reveals a more severe form of the disorder and offers fresh insights that could lead to improved treatment options.

 Cleft Lip Mystery Gets Molecular Clarity

Cleft Lip Mystery Gets Molecular Clarity

A molecular mechanism where impaired tRNA splicing disrupts cranial neural crest cells, revealing MYCN, DDX1, and tRNA processing defects as risk factors for orofacial clefts.

Return of Dire Wolves: Not Just in Game of Thrones, But in Real Life!

Return of Dire Wolves: Not Just in Game of Thrones, But in Real Life!

Genetic analysis reveals that dire wolves are not gray wolves’ ancestors but a separate species, reshaping evolutionary knowledge after 12,500 years.

 Faulty Gene May Raise Lung Risk in 1 in 3,000 People

Faulty Gene May Raise Lung Risk in 1 in 3,000 People

A faulty FLCN gene may affect 1 in 3,000 people, raising their risk of pneumothorax, but shows a much lower kidney cancer risk.

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