BioPharma, Hospitals

Fresh off first FDA approval, Amicus spends $100M to acquire 10 gene therapies

The company acquired 10 AAV-based gene therapies in development for lysosomal storage disorders, including two already in early clinical trials.

A drugmaker focused on rare, metabolic diseases is spending $100 million to acquire nearly a dozen clinical and preclinical gene therapies for lysosomal storage disorders developed by university researchers.

Amicus Therapeutics, based in Cranbury, New Jersey, said Thursday that it had acquired the 10 therapy programs, which were developed at the Center for Gene Therapy of the Research Institute at Nationwide Children’s Hospital and The Ohio State University. The programs are licensed to Amicus form Nationwide through the acquisition of Celenex, a spinout of the children’s hospital. Celenex shareholders are eligible for milestones of up to $277 million, for development and regulatory submissions in the US and Europe.

Amicus, which trades publicly on the NASDAQ under the ticker symbol FOLD and has a market cap of $2.3 billion, specializes in lysosomal storage disorders and has one commercial product, Galafold (migalastat), which the Food and Drug Administration approved last month for Fabry disease. It also has another drug in the clinic, AT-GAA, in Phase I/II development for Pompe disease.

The lead programs are for Batten disease – also known as neuronal ceroid lipofuscinosis, a rare, debilitating and potentially life-threatening disease – and include the clinical-stage assets CLN6 and CLN3, along with the preclinical drug CLN8. Other programs include therapies in development for Niemann Pick C, Wolman disease, Tay Sachs disease and multiple other central nervous system lysosomal storage disorders.

All of the gene therapies acquired under the Nationwide and Ohio State deal use the same adeno-associated viral vector approach. The first gene therapy to win FDA approval, Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl), for biallelic RPE65 mutation-associated retinal dystrophy, is also an AAV-based therapy, as are Spark’s other product candidates for other genetic diseases, along with those of competitor BioMarin. Meanwhile, bluebird bio is developing a lentiviral vector-based gene therapy, LentiGlobin, for the blood disorders beta-thalassemia and sickle cell disease.

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