Gene therapy appears to slow Huntington’s disease progression
On Sept. 24, 2025, a global clinical trial for a new Huntington’s disease treatment has posted positive results,…
gene therapy offers hope for genetic hearing loss
On Sept. 14, 2025, scientists from the Gray Faculty of Medical & Health Sciences at Tel Aviv University…
Biotechnology | Disease | FDA | Therapeutics
FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys; Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths
On Jul. 18, 2025, the U.S. Food and Drug Administration (FDA) announced it has placed Sarepta Therapeutics investigational…
CIRM awards $4.7 million for vision loss gene therapy
On May 29, 2025, The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated…
FDA Approved First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency
On Nov. 14, 2024, the U.S. Food and Drug Administration approved PTC Therapeutics’ Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated…
Gene therapy effective in hereditary blindness
On Sep. 10, 2024, researchers at the Karolinska Institute in Sweden announced a study that shows that gene…
Biotechnology | Diagnostics | Disease | FDA | Medical Device | Therapeutics
FDA Approved ‘First Gene Therapy’ to Treat Adults with Metastatic Synovial Sarcoma
On Aug. 2, 2024, the U.S. Food and Drug Administration approved Adaptimmune’s Tecelra (afamitresgene autoleucel), a gene therapy…
European Commission Approved Pfizer’s DURVEQTIX® (fidanacogene elaparvovec), a One-Time Gene Therapy for Adults with Hemophilia B
On Jul. 25, 2024, Pfizer announced that the European Commission (EC) has granted conditional marketing authorization for DURVEQTIX®…
CIRM awarded $26 million to advance clinical research across cancer, heart conditions, and rare diseases
On Jun. 28, 2024, the California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated…
Biotechnology | Disease | FDA | Medicine | Therapeutics
FDA Expanded Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy
On Jun. 20, 2024, the U.S. Food and Drug Administration expanded the approval of Sarepta Therapeutics’ Elevidys (delandistrogene…
Biotechnology | Diagnostics | Infectious Disease | Medicine | Neurology | Non-Profit Research | Oncology | Therapeutics
CIRM awarded $53 million to clinical research in heart failure and cystic fibrosis, supporting 10 translational projects
On May 30, 2024, the California Institute for Regenerative Medicine (CIRM) announced it had awarded $53 million to…
Biotechnology | Disease | FDA | Therapeutics
U.S. FDA Approved Pfizer’s BEQVEZ™ (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B
On Apr. 26, 2024, Pfizer announced that the U.S. Food and Drug Administration (FDA) had approved BEQVEZ™ (fidanacogene…
Biotechnology | Disease | FDA | Therapeutics
FDA approved first gene therapy for children with metachromatic leukodystrophy
On Mar. 18, 2024, the U.S. Food and Drug Administration (FDA) approved Orchard Therapeutics’ Lenmeldy (atidarsagene autotemcel), the…
Biotechnology | CRISPR | Disease | FDA | Therapeutics
CRISPR Therapeutics announced FDA approval of CASGEVY for treatment of ransfusion-dependent beta thalassemia
On Jan. 16, 2024, the U.S. Food and Drug Administration (FDA) announced it had approved CRISPR Therapeutics’ CASGEVY…
Biotechnology | Disease | FDA | Therapeutics
Swissmedic Authorises CSL’s HEMGENIX® (etranacogene dezaparvovec) as First Gene Therapy for Hemophilia B
On Jan. 15, 2024, CSL announced that Swissmedic had authorised HEMGENIX® (etranacogene dezaparvovec), the first and currently only gene…
CRISPR | Genomics | Stem Cell | Therapeutics
FDA approved first gene therapies to treat patients with sickle cell disease
On Dec. 8, 2023, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing…
bluebird bio announced commercial launch of LYFGENIA’ gene therapy for patients ages 12 and older with sickle cell disease
On Dec. 8, 2023, bluebird bio announced the U.S. commercial launch of its LYFGENIA’ (lovotibeglogene autotemcel, also known…
Vertex and CRISPR Therapeutics announced authorization of first CRISPR/Cas9 gene-edited therapy by UK MHRA for treatment of Sickle Cell Disease
On Nov. 16, 2023, Vertex and CRISPR Therapeutics announced that the United Kingdom (U.K.) Medicines and Healthcare products…
California’s Stem Cell Agency invested $44 million to advance regenerative medicine research and manufacturing in California
On Sept. 29, 2023, the California Institute for Regenerative Medicine (CIRM) awarded $43.8 million to fund projects aimed…
City of Hope received $32 million grant to advance novel therapies
On Aug. 24, 2023, researchers at City of Hope announced were awarded $32.3 million from the California Institute…
CIRM invested $50 million in clinical-stage research
On Jul. 27, 2023, the California Institute for Regenerative Medicine (CIRM) awarded $50.1 million to fund clinical-stage research…
Biotechnology | Diagnostics | Disease | FDA | Rare Disease | Therapeutics
U.S. FDA approved BioMarin’s ROCTAVIAN, the first and only gene therapy for adults with severe hemophilia A
On Jun. 29, 2023, the U.S. Food and Drug Administration approved Roctavian, an adeno-associated virus vector-based gene therapy…
Breakthrough Gene Therapy Offers Hope for Severe Developmental Epilepsy in Children
On Jun. 27, 2023, researchers from Tel Aviv University announced they have developed an innovative gene therapy that…
Biotechnology | Disease | FDA | Medicine | Therapeutics
U.S. FDA approved first gene therapy for treatment of patients with Duchenne Muscular Dystrophy
On Jun. 22, 2023, the U.S. Food and Drug Administration (FDA) approved Sarepta Therapeutics’s Elevidys, the first gene…
CIRM invested $10 million to build California Cell and Gene Therapy Manufacturing Network
On May 31, 2023, the California Institute for Regenerative Medicine (CIRM) awarded $10 million to five facilities as…
FDA approved first topical gene therapy for treatment of wounds in patients with dystrophic epidermolysis bullosa
On May 19, 2023, the U.S. Food and Drug Administration (FDA) approved Krystal Biotech’s Vyjuvek, a herpes-simplex virus…
Gene therapy allowed teen to see snowflakes for the first time
On Apr. 5, 2023, researchers at Oregon Health & Science University’s OHSU Casey Eye Institute announced that gene…
FDA approved first gene therapy for treatment of high-risk, non-muscle-invasive bladder cancer
On Dec. 16, 2022, the U.S. Food and Drug Administration approved Ferring Pharmaceuticals’ Adstiladrin (nadofaragene firadenovec-vncg), a non-replicating…
FDA approved first gene therapy to treat adults with Hemophilia B
On Nov. 22, 2022, the U.S. Food and Drug Administration approved the investigational gene therapy etranacogene dezaparvovec or…
First gene therapy for adults with severe hemophilia A, BioMarin’s ROCTAVIAN, approved by EC
On Aug. 24, 2022, BioMarin Pharmaceutical announced that the European Commission (EC) had granted conditional marketing authorization (CMA)…