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

Trending Activities

All Professions

More in Home

Basivertebral Nerve Ablation Beneficial for Chronic Back Pain

Basivertebral Nerve Ablation Beneficial for Chronic Back Pain

Improvement in self-reported outcomes at three months in patients with chronic lumbar back pain

FDA Approves Trulance for Chronic Idiopathic Constipation

FDA Approves Trulance for Chronic Idiopathic Constipation

Drug designed to stimulate secretion of intestinal fluid

ECG Could Be Used As Password for E-Health Record Access

ECG Could Be Used As Password for E-Health ...

Researchers say heartbeat could serve as a secure 'password' for patient's electronic medical history

is free, fast, and customized just for you!

Already a member?

Sign In Now »