Belief Biomed Congratulates Partner Askbio On IND Acceptance By FDA For Investigational Gene Therapy For Late-Onset Pompe Disease

Belief Biomed Congratulates Partner Askbio On IND Acceptance By FDA For Investigational Gene Therapy For Late-Onset Pompe Disease. SHANGHAI, Jan. 14, 2026 /PRNewswire/ -- AskBio Inc. (AskBio), a gene ...
pharmaphorum

FDA removes barriers to cell, gene therapy development

"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...

AskBio Announces FDA Acceptance of Investigational New Drug (IND) Application for AB-1009 Gene Therapy for Treatment of Late-Onset Pompe Disease (LOPD)

AB-1009 program advances to Phase 1/Phase 2; clinical trial initiated in the United StatesTherapy granted United States Food ...
Pharmaceutical Technology on MSN

FDA increases manufacturing flexibility for cell and gene therapies

Clinical and commercial quality controls are eased as the FDA looks to expedite therapy approvals in areas of unmet need.

Genprex Provides Clinical Update on Diabetes Gene Therapy Program

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

ALS gene therapy targeting TDP-43 protein put on FDA fast track

The FDA granted fast track status to VectorY’s VTx-002, an antibody gene therapy targeting TDP-43 protein aggregates in ALS.
GEN

Major Trends in Cell and Gene Therapy

As the CSO for genomic medicine at Cytiva, I have numerous interactions with our customers, and I attend many scientific and industry events. It’s great to be able to get first-hand exposure to what ...
STAT

Experimental gene therapy for Huntington’s markedly slowed disease progression in key trial

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast ...
CNN

What a $2 million per dose gene therapy reveals about drug pricing

This story was originally published by ProPublica. ProPublica is a nonprofit newsroom that investigates abuses of power. Sign up to receive its biggest stories as soon as they’re published. Vincent ...
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New gene therapy drugs can save lives, but many cost millions

A new class of drugs is saving the lives of children who once had no hope. These high-tech medicines can replace defective genes. But there's a catch. Many cost millions of dollars for a single dose.