Creative Biolabs is helping researchers overcome challenges of gene therapy research by providing analytical testing ...

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 use of ...

PDE6A-associated RP, a rare form of inherited retinal disease (IRD), is characterized by nyctalopia, visual field defects, and significant loss of visual acuity (VA). The disease primarily affects the ...

Tommy and Linda Pham founded RSF in 2021 to support R&D into a treatment for UBA5 disorder after their son, Raiden, was diagnosed with the extremely rare and progressive neurological condition. The ...

Researchers in China have reported a novel adeno-associated virus strategy that enables efficient in vivo reassembly and expression of large therapeutic genes, addressing a long-standing ...

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

A Phase 3 clinical trial testing SGT-003, a gene therapy candidate for Duchenne muscular dystrophy (DMD), will start dosing ...

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

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

Zhejiang Difference Biological Technology Co., Ltd. (DIFF Biotech) announced that its proprietary nasal spray influenza attenuated live vaccine, DIFF-flu, has recently received clinical trial approval ...

AskBio Inc., an RTP-based gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has received U.S. Food and Drug Administration (FDA) acceptance of its ...

Nine-year follow-up of gene therapy in haemophilia B confirms long-term safety and reduced bleeding, though full capsid ...