US-based biotechnology firm Amicus Therapeutics has entered into a definitive agreement to buy clinical stage gene therapy firm Celenex for $100m in an upfront payment.

The transaction will fetch Amicus worldwide development and commercial rights for ten gene therapy programmes from Nationwide Children’s Hospital, Ohio, US.

Amongst the programmes, CLN6, CLN3, and CLN8  are seen to have the potential to be first-to-market curative therapies for rare conditions such as Batten disease, which is oterhwise known Neuronal Ceroid Lipofuscinosis (NCL) .

All the ten gene therapy programmes leverage intrathecal delivery involving AAV vector approach, which is said to have been successfully used in clinical trials for some rare central nervous system (CNS) diseases.

Amicus Therapeutics chairman and CEO John Crowley said: “The in-licensing and acquisition of these gene therapy programmes provides an extraordinary opportunity to transform the lives of thousands of children living with some of the most devastating forms of lysosomal storage disorders, for which there are virtually no treatment options today.”

“Amicus is the optimal scientific and clinical partner to advance these programmes.”

The company intends to combine its expertise in global development and delivery of new therapies for rare metabolic disorders with Nationwide Children’s AAV technologies.

Nationwide Children’s Hospital Center for Gene Therapy principal investigator Kathrin Meyer said: “I firmly believe that Amicus is the optimal scientific and clinical partner to advance these programmes and look forward to actively collaborating with the Amicus team on the development of these important potential therapies and getting them to as many children as quickly as possible.”

In addition to the upfront payment, Celenex is eligible to receive up to $15m in development milestones and an additional $262m based on regulatory submission and approval milestones.

Amicus will pay up to $75m over the next four years in these milestones. The deal does not include royalties on any of the associated programmes.

However, Celenex shareholders may get up to $75m in tiered sales milestone payments.