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 ...

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 ...
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

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 ...

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 ...
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 ...

Regenxbio stock drops on FDA setback for RGX-121 gene therapy

Regenxbio (RGNX) shares fell after the U.S. Food and Drug Administration issued a complete response letter for RGX-121 ...
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 ...

Gene-editing therapy proves effective for Leber's hereditary optic neuropathy

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...
News Medical on MSN

Gene therapy targeting connexin 43 shows promise across inherited heart diseases

University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...