Gene Therapy Finds a Fertile Home in Ohio
Gene Therapy Finds a Fertile Home in Ohio
The midwestern state has quietly laid the groundwork for a biotech hub.
Gene Therapy Finds a Fertile Home in Ohio
Gene Therapy Finds a Fertile Home in Ohio

The midwestern state has quietly laid the groundwork for a biotech hub.

The midwestern state has quietly laid the groundwork for a biotech hub.

gene therapy
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
Lisa Winter | Jan 7, 2020
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.
Dog Study Revives Concerns About Virus Used for Gene Therapy
Dog Study Revives Concerns About Virus Used for Gene Therapy
Jef Akst | Jan 6, 2020
Canines treated with an adeno-associated viral (AAV) vector showed evidence that the therapeutic DNA held within the virus can integrate into the host genome, risking the activation of oncogenes.
Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
Alejandra Manjarrez | Dec 11, 2019
The animals lived longer and showed milder symptoms than untreated mice, although they didn’t survive as long as wildtype mice.
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Catherine Offord | Nov 12, 2019
The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.
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.