Ultragenyx to buy global rights to Abeona's gene therapy ABO-102 for fatal brain condition in kids

May 17, 2022 8:43 AM ETAbeona Therapeutics Inc. (ABEO), RAREBy: Ravikash, SA News Editor1 Comment

Luminous double helix strands of abstract DNA, 3D render.

ConceptCafe/iStock via Getty Images

  • Ultragenyx Pharmaceutical (NASDAQ:RARE) is acquiring global rights to Abeona Therapeutics' (NASDAQ:ABEO) AAV gene therapy ABO-102 (now UX111) to treat Sanfilippo syndrome type A (MPS IIIA).
  • Sanfilippo syndrome is a rare, fatal genetic condition that causes brain degeneration, and can show up in children by age three. This particular variant of the disease is caused by caused by genetic mutations that lead to a deficiency in the SGSH enzyme.
  • Under the the agreement, Ultragenyx will take responsibility for the ABO-102 program and in return Abeona is eligible to receive royalties of up to 10% on net sales and commercial milestone payments after regulatory approval.
  • "Our team’s expertise in MPS and gene therapy clinical development makes this program a seamless integration, and it has the potential to be our first gene therapy to market," said Ultragenyx CEO Emil Kakkis.
To ensure this doesn’t happen in the future, please enable Javascript and cookies in your browser.
Is this happening to you frequently? Please report it on our feedback forum.
If you have an ad-blocker enabled you may be blocked from proceeding. Please disable your ad-blocker and refresh.