Forbes

Gene Therapy: Revolutionizing Medicine By Tackling Diseases At Their Genetic Roots

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...
Labroots

Assessing Gene Therapy Approaches for Down Syndrome

Decades ago, researchers showed that Down syndrome (also known as trisomy 21), was caused when individuals carried an extra copy of chromosome 21. Down syndrome is also the most common genetic cause ...

Rare cranial disorders: Towards a non-invasive therapy using gene silencing delivered by nanoparticles and 3D printing

A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...

Adolore BioTherapeutics Announces FDA Orphan Drug Designation for its Kv7-activating rdHSV-CA8* Therapy in the Treatment of Erythromelalgia

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...

Belief BioMed Congratulates Partner AskBio on IND Acceptance by FDA for Investigational Gene Therapy for Late-Onset Pompe Disease

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, recently announced that the United States Food and Drug Administration (FDA) has ...
Forbes

Breakthrough Gene Therapy For Butterfly Children’s Disease

A revolutionary treatment brings new hope to those suffering from a devastating rare skin disorder, while showcasing an innovative funding model that could transform rare disease research In a ...
Benzinga.com

Abeona Therapeutics Wins FDA Approval For First-of-Its-Kind Gene Therapy

The U.S. Food and Drug Administration (FDA) on Tuesday approved Abeona Therapeutics Inc.’s (NASDAQ:ABEO) Zevaskyn (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the ...
Seeking Alpha

Fractyl Health Advances Rejuva® Gene Therapy Platform with Submission of First Clinical Trial Application Module in Europe for RJVA-001 in Type 2 Diabetes

BURLINGTON, Mass., May 19, 2025 (GLOBE NEWSWIRE) -- Fractyl Health, Inc. (GUTS) (the Company), a metabolic therapeutics company pioneering pattern-breaking treatments for obesity and type 2 diabetes ...
Ophthalmology Times

Ocugen publishes phase 1 GARDian1 gene therapy data for Stargardt disease

Ocugen has announced the publication of positive results from its phase 1 GARDian1 trial of OCU410ST, a gene therapy for ...
GEN

Precision Microfluidics for RNA and Gene Therapy Applications

As nucleic acid–based therapeutics evolve and mature, so too must the analytical tools that are designed to characterize them. New microfluidic capillary electrophoresis (CE) methods that are designed ...

Tessera Therapeutics Announces FDA Clearance of IND Application for its Lead In Vivo Gene Editing Program TSRA-196 for AATD

The Phase 1/2 trial is a first-in-human, open-label, multi-national study designed to evaluate the safety, tolerability, and efficacy of TSRA-196 in adults with AATD. Trial participants will receive a ...
FierceBiotech

uniQure's ballyhooed gene therapy for Huntington's hits FDA roadblock

The FDA is said to be making a U-turn on uniQure’s one-time gene therapy candidate for Huntington’s disease despite the treatment being hailed as a game changer. The FDA no longer agrees that data ...