FDA Halts Sarepta’s Gene Therapy Trials After Deaths; Platform Designation Revoked Amid Safety Concerns

The U.S. Food and Drug Administration announced it has placed Sarepta Therapeutics investigational gene therapy clinical trials for limb girdle muscular ...

July 21, 2025 | Monday | Regulatory
AAVantgarde Receives FDA Clearance for AAVB-039 to Treat Stargardt Disease

AAVantgarde Bio (AAVantgarde), a clinical-stage biotechnology company developing next-generation gene therapies for inherited retinal diseases,  ann...

July 16, 2025 | Wednesday | News
Ultragenyx Receives Complete Response Letter from FDA for UX111 Gene Therapy for Sanfilippo Syndrome Type A

Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its Biologics L...

July 14, 2025 | Monday | News
CARsgen Secures Final EPO Win for GPC3 CAR-T Patent After Appeal Withdrawal

CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, announces a favorable outco...

July 14, 2025 | Monday | News
Golden Age Health and Innostellar Sign Exclusive 10-Year Deal to Deliver Sight-Saving Gene Therapy LX-101 Across China

Golden Age Health Pte. Ltd. (“GAH”) and Innostellar Biotherapeutics Co., Ltd. (“Innostellar”)  announced an exclusive ten-ye...

July 14, 2025 | Monday | News
Purespring Therapeutics Receives FDA Clearance to Initiate Clinical Trials for IgA Nephropathy Gene Therapy

Purespring Therapeutics, a precision nephrology company focused on transforming the treatment of kidney diseases, announces that the U.S. Food and Drug A...

July 10, 2025 | Thursday | News
Longeveron Receives FDA Clearance to Begin Phase 2 Trial of Stem Cell Therapy for Pediatric Dilated Cardiomyopathy

 Longeveron Inc. (NASDAQ: LGVN), a clinical stage regenerative medicine biotechnology company developing cellular therapies for life-threatening and...

July 09, 2025 | Wednesday | News
Amarna Therapeutics and NorthX Biologics Advance Gene Therapy Manufacturing for Type 1 Diabetes

Amarna Therapeutics, (“Amarna” or “the Company”), a privately held biotechnology company pioneering transformative gene therapies...

July 09, 2025 | Wednesday | News
Orchard Therapeutics Completes Global Enrollment in Pivotal MPS-IH Gene Therapy Trial Ahead of Schedule

Orchard Therapeutics, a Kyowa Kirin company, announced the last patient has been treated in a registrational trial evaluating the efficacy and safety of ...

July 08, 2025 | Tuesday | News
PulseSight Therapeutics Doses First Patient in Phaase I Trial for First-in-Class Dry AMD Gene Therapy, PST-611

PulseSight Therapeutics SAS, an ophthalmology biotech company developing disruptive non-viral vectorized therapies with minimally-invasive delivery techn...

July 08, 2025 | Tuesday | News
ProBio Opens Flagship Cell and Gene Therapy Center of Excellence in New Jersey, Advancing Global Biomanufacturing Capabilities

ProBio, a global contract development and manufacturing organization (CDMO) specializing in cell and gene therapy, today announced the opening of its...

June 27, 2025 | Friday | News
Bristol Myers Squibb’s streamlined CAR T therapy labels signal broader patient access and regulatory confidence

Bristol Myers Squibb’s announcement that the US Food and Drug Administration has approved the removal of REMS programs an...

June 27, 2025 | Friday | News
Invetech and AiCella Forge Strategic Alliance to Accelerate AI-Driven Cell Therapy Manufacturing

Invetech, a global leader in solving complex manufacturing and product development challenges in the advanced therapies and diagnostics markets, today an...

June 27, 2025 | Friday | News
AGC Biologics Expands Global Cell Therapy Network with New Services Launch in Yokohama, Japan

AGC Biologics, your friendly CDMO expert, will commence cell therapy process development and clinical manufacturing services on July 1, 2025, at AGC Inc....

June 26, 2025 | Thursday | News

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