New Scientist

First ever inhalable gene therapy for cancer gets fast-tracked by FDA

A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...
BioSpace

REGENXBIO’s Hunter Syndrome Gene Therapy Fails To Win FDA Nod

The FDA recommended that REGENXBIO run a new study, treat more patients and include a placebo arm to support a resubmission ...
Fierce Biotech

FDA rejects Regenxbio's gene therapy in Hunter syndrome, leaving CEO 'concerned'

After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by ...
Targeted Oncology

FDA Grants RMAT Designation to Inhalable Gene Therapy for Advanced Lung Tumors

FDA RMAT boosts inhaled gene therapy KB707, delivering IL‑2/IL‑12 to lung tumors; early trial shows responses with manageable ...

US FDA declines to approve Regenxbio's gene therapy for rare disease

Feb 9 (Reuters) - Regenxbio said on Monday the U.S. Food and Drug Administration had declined to approve its gene therapy for ...
Fierce Biotech

Lilly drops 3 pipeline drugs, including gene therapy from $1B Prevail buyout

As Eli Lilly awaits an FDA approval decision for its oral GLP-1 orforglipron, the pharma has discarded three clinical-stage ...
BioPharma Dive

FDA rejects Regenxbio treatment in another blow to gene therapy

The decision comes two weeks after the agency halted testing due to safety concerns and represents the latest regulatory ...

Genprex Announces IP Australia's Intent to Grant Patent for Reqorsa® Gene Therapy in Combination with PD-L1 Antibodies to Treat Cancers

Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing ...
MedPage Today on MSN

Gene therapy shows signs of delayed progression in fatal neurodegenerative disease

All participants had adverse events, but most were moderate in severity ...
BioSpace

Ultragenyx’s Gene Therapy Shows Long-Term Efficacy in Two Sanfilippo Studies

The FDA in July last year declined to approve UX111 for Sanfilippo syndrome, a rare neurodegenerative disorder, citing ...
Monitor

New gene therapy offers hope to sickle cell patients

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...
News Medical on MSN

First topical CRISPR gene therapy corrects disease-causing mutations in human skin

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the University of British Columbia are extending those possibilities to the skin for ...