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 ...
Interesting Engineering on MSN

First-ever experimental gene therapy seeks to restore vision by rejuvenating eye neurons

Life Biosciences is pushing cellular rejuvenation into the clinic with ER-100, an experimental gene ...
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 ...
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 ...
Hoodline

South Jersey Sickle Cell Sufferer Says One-Shot Gene Fix Erased His Pain

For most of his 20 years, a New Jersey man knew pain as a daily reality. Now, after a one-time CRISPR gene-editing treatment at Children's Hospital of Philadelphia, Austin Louis says he is finally ...

Apertura Gene Therapy Launches Open Aperture, a Material Transfer Agreement Program to Facilitate Non-Commercial Access to TfR1 CapX for Academic Research…

Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, today ...
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 ...

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 ...
BioSpace

Vertex, CRISPR Set Lofty Goal for Casgevy Gene Therapy as Patient Starts Ramp

Following over a year of slow uptake, Vertex Pharmaceuticals and CRISPR Therapeutics expect Casgevy revenues to nearly triple ...
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 ...

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

Global trends shape progress in cell and gene therapies

Cell and gene therapies , or CGT , have come a long way since they were first introduced. In the last few decades, both cell therapy -- the transplantation of living cells -- and gene therapy -- the ...