Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold

The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.

The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.

gene therapy
Google Bans Ads for Experimental Medical Treatments
Google Bans Ads for Experimental Medical Treatments
Ashley Yeager | Sep 9, 2019
The new policy will increase scrutiny and pressure on stem cell clinics, according to industry experts.
Leaders of AveXis Out Over Doctored Gene Therapy Data
Leaders of AveXis Out Over Doctored Gene Therapy Data
Kerry Grens | Aug 14, 2019
Novartis, which bought the biotech firm in 2018, announced that the company’s top scientists have left, and news reports say it’s because they were involved in using manipulated data to get the gene therapy Zolgensma approved.
Immune-Activating Gene Therapy for Glioblastoma
Immune-Activating Gene Therapy for Glioblastoma
Ruth Williams | Aug 14, 2019
The results of an early trial in 31 brain cancer patients finds immune activity boosted in the tumor, and possibly longer survival.
Some Data Backing Novartis Gene-Therapy Approval Manipulated: FDA
Some Data Backing Novartis Gene-Therapy Approval Manipulated: FDA
Nicoletta Lanese | Aug 7, 2019
The agency is now evaluating the implications of the corrupted data behind Zolgensma and whether to “take action” against the pharmaceutical company.
Researchers Launch First Study of In Vivo CRISPR Therapy in Humans
Researchers Launch First Study of In Vivo CRISPR Therapy in Humans
Catherine Offord | Jul 26, 2019
A trial is recruiting patients to test the gene-editing technology’s ability to treat an inherited form of blindness caused by a mutation in the CEP290 gene.
Strategies for Smuggling Gene Therapies Past the Immune System
Strategies for Smuggling Gene Therapies Past the Immune System
Monique Brouillette | Jul 15, 2019
Researchers are devising ways to prevent viral vectors carrying gene therapies from triggering an immune response.
Stem Cell Funding Agency CIRM Is Nearly Out of Funds
Stem Cell Funding Agency CIRM Is Nearly Out of Funds
Chia-Yi Hou | Jul 8, 2019
The California Institute for Regenerative Medicine has been funding research since it formed in 2004, giving out nearly $3 billion in grants to date.
FDA Approves Gene Therapy for Spinal Muscular Atrophy
FDA Approves Gene Therapy for Spinal Muscular Atrophy
Ashley Yeager | May 27, 2019
At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.
Image of the Day: Cone Preservation
Image of the Day: Cone Preservation
Chia-Yi Hou | Apr 30, 2019
Researchers treat mice with retinitis pigmentosa using a gene therapy that reduces cone loss.