Rare Disease Report

Abeona Granted $13 Million to Continue Development of MPS III Gene Therapies

OCTOBER 16, 2017
Mathew Shanley
On Monday morning, Abeona Therapeutics announced that the company was a recipient of $13.85 million in grants intended to fund the advancement of clinical stage gene therapies for 2 variations of mucopolysaccharidosis (MPS), or Sanfilippo disease.

MPS is a group of metabolic disorders caused by the absence or improper functionality of lysosomal enzymes necessary to break down long chains of sugar carbohydrates called glycosaminoglycans. These enzymes are partly responsible for the building of bone, cartilage, tendons, corneas, skin, and connective tissue.

MPS type IIIA and type IIIB are more common than types IIIC and IIID, however, there are very few differences between the 4 variations. In all cases, patients with the condition are unable to break down sugar molecules, however, patients with the B form can remain relatively healthy into adult life.

There are currently no effective treatments for MPS of any kind, however, Abeona recently revealed that positive responses were elicited in 1 year data from Cohort 1 of a 3-cohort, Phase 1/2 trial evaluating a single intravenous injection of ABO-102 (AAV-SGSH).

The company is also developing ABO-101 (AAV-NAGLU) for MPS IIIB.

“Abeona is pleased to continue our global collaboration with the Sanfilippo foundations to help further advance our gene therapy programs for MPS III disease,” said Timothy J. Miller, Ph.D., president and chief executive officer of Abeona Therapeutics in a press release. “The effort and expertise that we continue to commit to the ABO-102 and ABO-101 programs puts us in a strong position to further extend the important progress reported to date.  We are grateful to the foundations for their ongoing commitment to identifying and facilitating the development of clinical innovation to treat patients with MPS III disease.”

The 9 companies contributing to the grant are: Team Sanfilippo Foundation, Stop Sanfilippo Fundación, Fundación Sanfilippo B, Sanfilippo Children's Foundation, the National MPS Society, the Red Sanfilippo Foundation, the Children's Medical Research Foundation, and the Abby Grace Foundation and Fondation Sanfilippo Suisse.

“The importance of reducing the heparan sulfate as a cause of disease burden cannot be understated, and the clinical data demonstrated by Abeona enabled us to provide additional support in the pursuit of finding new paradigms to treat all children with Sanfilippo syndrome,” stated Carl Kapes, Board Member of Team Sanfilippo.

In addition to the collaboration grant, Abeona is receiving an infusion of an additional $5 million from exercise of outstanding warrants, totaling $18.85 million in proceeds.

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