Rare Disease Report

Trial for Gene Therapy to Treat Sanfilippo Begins in Spain

NOVEMBER 10, 2017
Mathew Shanley
Abeona Therapeutics announced today that the first patient in Spain was enrolled in its Phase 1/2 clinical trial for ABO-102 (AAV-SGSH) for the treatment of Sanfilippo syndrome Type A (mucopolysaccharidosis type IIIA [MPS IIIA]).

In October, one year data from Cohort 1 (1x dose escalation) of the trial was released that showed ABO-102 elicited positive responses in patients, and a week later, the first subject in Cohort 3 (6x dose escalation) was enrolled at Adelaide Women’s and Children’s Hospital in Adelaide, Australia. Through 30 days post-injection, the patient exhibited significant reductions of cerebral spinal fluid (CSF) heparin sulfate (HS) and urinary HS.

HS, a large sugar molecule, is unable to be broken down in patients with Sanfilippo syndrome, which can lead to symptoms like progressive dementia, aggressive behavior, hyperactivity, and seizures.

The recently-enrolled patient is a part of Cohort 3 at the Hospital Clinico Universitario of Santiago de Compostela, Spain.

"We are pleased to initiate enrollment at our Spain clinical site for ABO-102.  We remain encouraged by the improvements observed in clinically relevant biomarkers post-dosing of the gene therapy in the patients in Cohort 3 and the ongoing safety profile ABO-102 demonstrates,” stated Juan Ruiz, M.D., Ph.D., Chief Medical Officer of Abeona Therapeutics in a press release.

Nine total subjects have been infused in the dose-escalation trial thus far, equaling more than 2,000 cumulative follow-up days.

“Developing a local company presence in Spain allows us to exercise closer supervision and further advance Abeona’s gene therapy programs in Europe, as well as advance our relationships with the patient community,” said Ruiz. “We are grateful to the many patient foundations and parents who have supported the research needed to advance a potential treatment for this devastating unmet medical need. We are also proud to collaborate with one of the leading clinical centers in Spain dealing with MPS IIIA patients.”

Subjects in the ongoing study receive a single, intravenous injection of ABO-102 to deliver the adeno-associated virus (AAV) viral vector systemically throughout the body with the intention of introducing a corrective copy of the underlying gene that causes the condition.

The year-one data released in October exhibited that ABO-102 is well-tolerated at all doses and follow-up timeframes, and that the drug provides a clear clinical benefit.

“It’s really important for the communities that we keep an open channel of communication with patients and families so that they know where we are conducting these studies,” said Timothy Miller, PhD, CEO of Abeona Therapeutics. “You find many parent-led organizations that try to work within an umbrella structure, and we feel as though we’re fulfilling our promises to them that we are coming, and we are striving to find new ways to treat your kids. When there’s nothing else out there, this information is the lifeline that a lot of these people have.”

For more information from Abeona and the ABO-102 trials that are ongoing, follow Rare Disease Report on Facebook and Twitter.

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