Medical Professional Publications

FDA Approves Clinical Trial for Sanfilippo Syndrome Type A, Gene Therapy Developed by Researchers at Nationwide Children's Hospital

(From the April 2016 issue of Research Now)

February 29, 2016 was a very big day for gene therapy researchers at Nationwide Children’s Hospital and for patients with Sanfilippo syndrome type A, a rare and life-threatening genetic disease that leads to children to progressively lose the ability to speak, walk and eat before their eventual deaths.

Sanfilippo syndrome type A is the most common and severe type of Sanfilippo syndrome, occurring in 1 in every 70,000 births. Children appear typical at birth and usually do not show symptoms until after their first year of life. Currently, there is no cure for Sanfilippo syndrome, and treatments mainly include supportive care.

But on February 29, Abeona Therapeutics, a company that focuses on developing treatments for severe and life-threatening rare diseases, announced FDA approval of a new clinical trial for Sanfilippo syndrome type A. The trial will use a gene therapy approach, also known as ABO-102, developed by researchers at Nationwide Children’s.

“The progress of ABO-102 into human clinical trials represents a significant milestone in advancing our rare diseases product pipeline towards achieving value for patients and their families, as well as shareholders,” states Steven Rouhandeh, executive chairman of Abeona Therapeutics.

Douglas McCarty, PhDHaiyan Fu, PhD, and Kevin Flanigan, MD, are principal investigators in the Center for Gene Therapy at The Research Institute at Nationwide Children’s who developed the novel gene therapy approach, which involves a one-time delivery of a normal copy of the defective gene to cells of the central nervous system, to reverse the effects of the genetic errors that cause the disease.

“The gene therapy approach developed by Drs. Douglas McCarty and Haiyan Fu at Nationwide Children’s represents a new treatment paradigm for addressing this relentlessly progressing disease with a single, intravenous administration,” says Dr. Flanigan. “We would like to thank these dedicated researchers, as well as the many patient foundations, for their dedicated support and commitment to advancing new treatment options forward for this devastating unmet medical need.”

The FDA-approved study is a phase 1/2 dose escalation clinical trial, which means it is designed to find the best and safest dose based on how well patients can tolerate the treatment. The trial will include six to nine patients with Sanfilippo syndrome type A.

Full news release:

Abeona Therapeutics Announces FDA Allowance of Investigational New Drug (IND) for systemic AAV Phase 1/2 Clinical Study with AB0-102 Gene Therapy for Patients with Sanfilippo syndrome type A (MPS IIIA)

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