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BMN 044

BioMarin Pharmaceutical Inc.

Drug Names(s): PRO044

Description: PRO044 is highly sequence-specific, specifically induces exon 44 skipping in the duchenne muscular dystrophy (DMD) gene. This is applicable to 6% of all DMD patients, including patients with deletions of exon 43, exon 45, exons 38-43, exons 40-43, exons 42-43, and exons 45-54. The underlying chemistry and mechanism are similar to PRO051. Along with PRO051, PRO044 is extensively tested in vitro in series of cultured muscle cells from patients with different relevant mutations, and in the hDMD mouse model.

Deal Structure: Prosensa and GSK
In October 2009, Prosensa and GlaxoSmithKline (GSK) announced that they entered into an exclusive worldwide collaboration for the development and commercialization of RNA based therapeutics for Duchenne Muscular Dystrophy (DMD).

In September 2011, Prosensa announced that they have agreed with GlaxoSmithKline (GSK) to advance three further exon skipping compounds (PRO044, PRO045 and PRO053) into the next development stage under their ongoing collaboration relationship in Duchenne Muscular Dystrophy (DMD). Under the collaboration agreement, Prosensa is eligible for milestone payments and development funding payments which could total up to GBP 27M, depending upon the course and outcome of the clinical development for these programs. Under the terms of the alliance established in 2009, GSK has an exclusive option to licence PRO044 and an option to licence one of either PRO045 or PRO053 for later stage development and commercialisation, with Prosensa...See full deal structure in Biomedtracker

BMN 044 News

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