ASH: Single Dose of Gene Therapy Effective in Hemophilia B

Share this content:
ASH: Single Dose of Gene Therapy Effective in Hemophilia B
ASH: Single Dose of Gene Therapy Effective in Hemophilia B

THURSDAY, Dec. 8, 2016 (HealthDay News) -- A single dose of an experimental gene therapy may benefit patients with hemophilia B, according to trial results presented at the annual meeting of the American Society of Hematology, held from Dec. 3 to 6 in San Diego.

In the combined phase 1/phase 2 trial, researchers gave a single dose of the gene therapy to nine adult hemophilia B patients who ranged in age from 18 to 52.

The patients' levels of factor IX rose to a level of around 30. Patients who got the gene therapy were able to stop getting infusions of the clotting factor.

"At these new levels, hemophilia patients do not typically need to self-treat with factor [IX] to avoid bleeding events," lead investigator Lindsey George, M.D., hematologist at Children's Hospital of Philadelphia, said in a hospital new release. "This represents a potential dramatic improvement in their quality of life and a shift in the way we think about treating hemophilia."

Children's Hospital of Philadelphia has a financial interest in a gene therapy company that spun off from the hospital. The senior author of the study was formerly a researcher at the hospital and now is president and chief scientific officer of the company.

Press Release
More Information

Share this content:

is free, fast, and customized just for you!

Already a member?

Sign In Now »


Sign up for myCME e-newsletters


Drug Lookup

Browse drugs by: BrandGenericDisease

More in Home

Illicit Marijuana Use Up in States With Legal Medical Use

Illicit Marijuana Use Up in States With Legal ...

Cannabis use disorder is also rising faster in states with legalization laws

Chromosome Instability Test Prognostic in NSCLC

Chromosome Instability Test Prognostic in NSCLC

Chromosome instability associated with higher risk of relapse, death within 2 years

CDC: Opioid-Related Mortality Rate Might Be Underestimated

CDC: Opioid-Related Mortality Rate Might Be Underestimated

Death certificates from drug-linked infections may not label opioids as possible cause

is free, fast, and customized just for you!

Already a member?

Sign In Now »