Dog Study Revives Concerns About Virus Used for Gene Therapy
Dog Study Revives Concerns About Virus Used for Gene Therapy
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.
Dog Study Revives Concerns About Virus Used for Gene Therapy
Dog Study Revives Concerns About Virus Used for Gene Therapy

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.

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.

clinical trials
FDA Approves Previously Rejected Muscular Dystrophy Treatment
FDA Approves Previously Rejected Muscular Dystrophy Treatment
Emily Makowski | Dec 13, 2019
The drug Vyondys 53 has met a surrogate endpoint of protein production, but there’s no clear proof it has a clinical benefit.
Biogen Presents Data on Efficacy of Alzheimer’s Drug
Biogen Presents Data on Efficacy of Alzheimer’s Drug
Shawna Williams | Dec 6, 2019
The company and its partner halted two clinical trials for futility early this year, but now say they’ll seek approval for aducanumab.
Interactive: Biomarkers in Blood Provide a Window into the Brain
Interactive: Biomarkers in Blood Provide a Window into the Brain
Shawna Williams | Dec 1, 2019
A look at some of the circulating molecules that may indicate various Alzheimer’s pathologies and serve as the bases of noninvasive tests for the disease.
The Hunt for a Blood Test for Alzheimer’s Disease
The Hunt for a Blood Test for Alzheimer’s Disease
Shawna Williams | Dec 1, 2019
Researchers hope circulating biomarkers will enable earlier detection and better monitoring of the neurodegenerative disorder—and perhaps help usher in new treatments.
Early Results Are Positive for Experimental CRISPR Therapies
Early Results Are Positive for Experimental CRISPR Therapies
Jef Akst | Nov 19, 2019
Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.
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.
Neural Stem Cell Transplantation Crawls Toward the Clinic
Neural Stem Cell Transplantation Crawls Toward the Clinic
Ashley P. Taylor | Oct 29, 2019
Several early-stage clinical trials indicate that implanting patients with the cells is safe. But whether they can alleviate neurological problems remains to be seen.
UCB Cuts $2.1 Billion Deal to Acquire Ra Pharma
UCB Cuts $2.1 Billion Deal to Acquire Ra Pharma
Catherine Offord | Oct 14, 2019
The move will allow the Belgium-headquartered biopharmaceutical giant to boost its work on treatments for rare diseases.
Biogen, Eisai End Two Late-Stage Trials for Alzheimer’s Treatment
Biogen, Eisai End Two Late-Stage Trials for Alzheimer’s Treatment
Emily Makowski | Sep 16, 2019
The widely expected move comes months after the companies scrapped trials of another Alzheimer’s drug.