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Doctors, Heal Infant and Gene Editing

Breakthrough Gene Therapy Saves Infant with Rare Metabolic Disease

A personalized gene editing therapy was successfully used to treat an infant with CPS1 deficiency, a disease that causes ammonia buildup in the blood. The technology used creates an opportunity for the development of personalized treatments for other rare diseases.

Managed Healthcare Executive · 4h

· 1d · on MSN

Doctors Achieve Medical Breakthrough Using Gene-Editing Therapy to Heal Baby with Rare Disorder

· 3d

Doctors Heal Infant Using First Customized-Gene Editing Treatment

Fierce Biotech1h

Atsena eye disease gene therapy hits safety goals, closes retinal splits in phase 1/2

Hot on the heels of a $150 million fundraise, Atsena Therapeutics is back with early-stage data for the company’s eye disease ...

Medera Showcased Positive Interim Data From First-in-Human Gene Therapy Trial for HFpEF in Late-Breaking Presentation at Heart Failure 2025 Congress

Results from MUSIC-HFpEF Phase 1/2a clinical trial show favorable safety profile and early clinical benefits with SRD-002 ...

14hon MSN

SA family of 4-year-old battling terminal disease asks for community support for gene therapy trial

A gene therapy trial in California at the end of the year has the potential to greatly impact Charli's quality of life.

BioSpace5h

ASGCT Roundup: Sarepta, Rocket, Neurogene and the World’s First Tailored CRISPR Therapy

Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a ...

Discover Magazine on MSN2d

CRISPR Fulfills Its Promise with First-Ever Personalized Gene-Editing Therapy

When CRISPR was first introduced as a gene-editing tool in 2012, the world was in awe of all the possibilities it held - ...

From birth to gene-edited in 6 months: Custom therapy breaks speed limits

The researchers described the unprecedented speed of the oversight steps as being "through alternative procedures." In month ...

Fierce Biotech3d

ASGCT: Analysts see Rocket gene therapy setting 'a new bar' for efficacy in heart condition

Rocket Pharmaceuticals has shared early data from three patients that tie its gene therapy for an inherited heart disease to ...

Science Daily3d

Gene Therapy News

Infant With Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment May 15, 2025 — A research team has developed and safely delivered a personalized gene ...

5hon MSN

SA family of 4-year-old girl with terminal disease, needs community's support for gene therapy trial

SAN ANTONIO — Charli is fighting an extremely rare and terminal condition often referred to as childhood dementia at just ...

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