Science & Technology

Amicus Therapeutics Acquires Gene Therapy Portfolio

Amicus Therapeutics announced the signing of a definitive agreement in which Amicus Therapeutics will receive worldwide development and commercial rights for ten gene therapy programs developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University. The 10 programs are licensed to Amicus from Nationwide Children’s Hospital through the acquisition of Celenex, a private, clinical stage gene therapy company. The lead programs in CLN6, CLN3, and CLN8 Batten disease are potential first-to-market curative therapies for these rare, devastating diseases. Batten disease, also known as Neuronal Ceroid Lipofuscinosis (NCL), is a family of rare disorders that can be life-threatening and debilitating, with high unmet medical need. More detail about these programs will be presented on this morning’s conference call. The slides for this conference call are available on our website.

“The in-licensing and acquisition of these gene therapy programs 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,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics. “The groundbreaking work of Drs. Brian Kaspar and Kathrin Meyer at Nationwide Children’s Hospital, along with collaborator, Arthur Burghes, Ph.D., professor at The Ohio State University, on these programs has led to remarkably strong and consistent pre-clinical results and now, in CLN6 Batten disease, encouraging early results in children. This is science and biotechnology at its best. And it has at its core the love, drive and passion of two remarkable parents, Gordon and Kristen Gray, who moved heaven and earth to partner with these researchers to advance these potentially life-saving medicines for their daughters and now for many thousands more. I am honored that they and their research team have chosen to entrust these ten programs to the passionate team of scientists and entrepreneurs at Amicus. I cannot think of a better foundation for Amicus’ entry into gene therapies.”

All acquired programs leverage intrathecal delivery, using the same AAV vector approach utilized successfully in clinical trials across other rare CNS indications, such as SMA. This approach and technology are considered to be a clinically validated gene delivery platform for diseases of the central nervous system (CNS). Brian Kaspar, Ph.D., co-founder of Celenex, and Kathrin Meyer, Ph.D., a Principal Investigator at Nationwide Children’s Hospital Center for Gene Therapy, will continue to support these programs as scientific advisors to Amicus Therapeutics.

“The preclinical proof-of-concept we have seen to date in CLN6, CLN3, and CLN8 further support the applicability of the AAV vector we developed at Nationwide Children’s in genetic disease of the CNS,” said Kathrin Meyer, Ph.D. “I firmly believe that Amicus is the optimal scientific and clinical partner to advance these programs 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. They truly have the potential to transform lives.”

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