Sarepta Therapeutics to acquire global rights to four clinical and three preclinical programs from Arrowhead Pharma
Arrowhead Pharmaceuticals granted Sarepta Therapeutics global rights on 26 November to four clinical and three preclinical programs in rare, genetic diseases of the muscle, central nervous system and the lungs
In addition, the pact will allow Sarepta to select up to six new targets for Arrowhead to conduct discovery and preclinical development activities in other rare disease areas. The clinical programs use Arrowhead’s Targeted RNAi Molecule (TRiM) platform, designed to deliver small interfering RNA (siRNA) to multiple tissue and cell types throughout the body to initiate the RNA-interference mechanism and induce rapid and durable knockdown of target genes. Clinical-stage candidates licensed under the deal include: ARO-DUX4, which targets the gene that encodes the DUX4 protein as a potential treatment for patients with facioscapulohumeral muscular dystrophy type 1 (dosing patients in a Phase I/II trial); ARO-DM1, which reduces expression of the dystrophia myotonica protein kinase (DMPK) gene in skeletal muscle as a potential treatment for patients with type 1 myotonic dystrophy (dosing patients in Phase I/II); ARO-MMP7, which reduces expression of matrix metalloproteinase 7 (MMP7) in pulmonary epithelial cells as a potential treatment for idiopathic pulmonary fibrosis (dosing patients in Phase I/II); and ARO-ATXN2, which is designed to silence expression of the toxic ATXN2 protein in the central nervous system as a potential treatment for spinocerebellar ataxia 2 (SCA2) (Phase I/II). Preclinical programs include ARO-HTT for Huntington’s disease, ARO-ATXN1 for spinocerebellar ataxia 1 (SCA1), and ARO-ATXN3 for spinocerebellar ataxia 3 (SCA3).
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