It’s time for regulatory agencies, academics and pharmaceutical companies to convene to refine guidance for long-term follow-up of patients receiving gene therapy. That was the message Anne-Virginie ...
Ocugen, Inc. announced its participation in the 2025 BIO International Convention in Boston, taking place from June 16-19. The company will present their innovative modifier gene therapy platform that ...
European Medical Journal: CRISPR Gene Therapy Shows Promise in Sickle Cell Disease
CRISPR gene therapy increases haemoglobin and fetal haemoglobin in sickle cell disease, reducing vaso occlusive events in early clinical data.
A CRISPR Therapy Just Cured A Disease From Inside The Body For The First Time. The Gene-Editing Era Officially Started Monday.
The idea behind intra-articular gene therapy for treating osteoarthritis (OA) is to deliver the gene-altering vector or cells directly to the precise site of the disease with a single injection — so ...
Oregonian: Gene editing helps a baby with a rare disease. Doctors hope it will help millions someday
Gene editing helps a baby with a rare disease. Doctors hope it will help millions someday
Forbes: New Gene Therapy For Rare Hearing Loss Wins FDA Approval—What To Know
New Gene Therapy For Rare Hearing Loss Wins FDA Approval—What To Know
The Irish Times: Irish baby among world’s first to receive experimental gene therapy
An Irish child is among a small group whose progress is being monitored internationally after receiving a potentially life-saving experimental gene therapy in the United States. It was not a move to ...
Reuters: CRISPR gene therapy slashes 'bad' cholesterol, triglycerides by half in small study
CHICAGO, Nov 8 (Reuters) - A single infusion of CRISPR Therapeutics' (CRSP.BN), opens new tab experimental gene therapy was safe and reduced levels of harmful LDL cholesterol and triglycerides by half ...
CRISPR gene therapy slashes 'bad' cholesterol, triglycerides by half in small study
The New England Journal of Medicine: AAV9 Gene Therapy in Type II GM1 Gangliosidosis — A Phase 1–2 Trial
AAV9 Gene Therapy in Type II GM1 Gangliosidosis — A Phase 1–2 Trial
The Manila Times: Fractyl Health Authorized to Initiate First-in-Human Trial of RJVA-001 in the Netherlands: First Gene Therapy Candidate to Enter Clinical Development for Type 2 Diabetes
Fractyl Health Authorized to Initiate First-in-Human Trial of RJVA-001 in the Netherlands: First Gene Therapy Candidate to Enter Clinical Development for Type 2 Diabetes
GEN - Genetic Engineering and Biotechnology News: Gene Therapy ETX101 Improves Seizures and Neurodevelopment in Dravet Syndrome in Phase I/II
New POLARIS Phase I/II data presented at ASGCT show ETX101 gene therapy reducing seizures and improving neurodevelopment in children with SCN1A+ Dravet syndrome.
Gene Therapy ETX101 Improves Seizures and Neurodevelopment in Dravet Syndrome in Phase I/II
FierceBiotech: Regenxbio posts clean safety profile for DMD gene therapy as pivotal data loom
Regenxbio has guided its Duchenne muscular dystrophy (DMD) gene therapy candidate through another test, reporting a clean safety profile and functional improvements as it heads toward pivotal data.
Regenxbio posts clean safety profile for DMD gene therapy as pivotal data loom
MedCity News: A New Era for Cell and Gene Therapy Signals New Market Opportunities
A New Era for Cell and Gene Therapy Signals New Market Opportunities
TMCnet: Addition Therapeutics Presents Preclinical Proof-of-Concept Data at American Society of Gene & Cell Therapy (ASGCT) 29th Annual Meeting
Addition Therapeutics Presents Preclinical Proof-of-Concept Data at American Society of Gene & Cell Therapy (ASGCT) 29th Annual Meeting
Markets Insider: AskBio Announces 6 Presentations at American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting
AskBio Announces 6 Presentations at American Society of Gene and Cell Therapy (ASGCT) 29th Annual Meeting
Times Now on MSN: FDA Approves First-Ever Gene Therapy for Hearing Loss
FDA approves Otarmeni gene therapy for genetic hearing loss; Regeneron offers free treatment, restoring hearing by correcting OTOF mutations.
PharmiWeb: Skylark Bio to Present New Preclinical Data for its Pendrin Gene Therapy Program for Hearing Loss at ASGCT 2026
Skylark Bio to Present New Preclinical Data for its Pendrin Gene Therapy Program for Hearing Loss at ASGCT 2026
FiercePharma: Gene therapy CDMO Artis BioSolutions launches with acquisition of Landmark Bio
Pharmaceutical Technology on MSN: Latus Bio secures $97M in funding to boost gene therapy access
MedCity News: Cash-Strapped Gene Therapy Firm Bluebird Bio Agrees to Acquisition by Private Equity
Cash-Strapped Gene Therapy Firm Bluebird Bio Agrees to Acquisition by Private Equity
PharmiWeb: Latus Bio Announces $97 Million Series A Financing to Expand the Reach of Gene Therapy to Larger Populations
Latus Bio Announces $97 Million Series A Financing to Expand the Reach of Gene Therapy to Larger Populations
FinanzNachrichten.de: Viralgen Partners with Elaaj Bio to Advance Gene Therapy Program for CDKL5 Deficiency Disorder
Collaboration leverages AAV manufacturing expertise to advance a preclinical-stage gene therapy program for a rare pediatric neurological disorder.SAN SEBASTIÁN, Spain and LONDON, ...
Viralgen Partners with Elaaj Bio to Advance Gene Therapy Program for CDKL5 Deficiency Disorder
Nasdaq: Ocugen, Inc. to Present at 2025 BIO International Convention, Showcasing Gene Therapy Advancements for Blindness Diseases
Ocugen, Inc. to Present at 2025 BIO International Convention, Showcasing Gene Therapy Advancements for Blindness Diseases
We define a gene as a DNA sequence that is transcribed. This definition includes genes that do not encode proteins (not all transcripts are messenger RNA). The definition normally excludes regions of the …
gene, unit of hereditary information that occupies a fixed position (locus) on a chromosome. Genes achieve their effects by directing the synthesis of proteins. In eukaryotes (such as animals, …
Genes are segments of DNA that give your cells instructions for specific traits or body functions. Different versions of the same gene are called alleles.
A gene is a specific stretch of DNA that carries the instructions for making one particular protein or performing one particular function. Humans have roughly 19,400 protein-coding genes, but …
Genes are specific sections of DNA that have instructions for making proteins. Proteins make up most of the parts of your body and make your body work the right way. You have two copies of every gene. You …
A gene is a segment of DNA that contains instructions for building one or more molecules that help the body work. Researchers estimate that humans have about 20,000 genes, which account …