Money will go toward development of compounds for skipping of exon 52.

Prosensa, a biopharmaceutical company focusing on RNA modulating therapeutics for rare diseases, received $1.5 million from Charley’s Fund. The financing will support development of compounds for the skipping of exon 52 in patients with Duchenne muscular dystrophy (DMD).

Under terms of the agreement, the funding will span three years. Prosensa will work to identify antisense oligonucleotides for the skipping of exon 52 in specific subpopulations of DMD patients. Prosensa will assess the safety and efficacy of the compounds in suitable preclinical models.

“Since our first investment in Prosensa six years ago, we have been impressed by their steadfast progress in drug development for DMD,” says Benjamin D. Seckler, M.D., founder and president of Charley’s Fund.

“Working closely with DMD patient advocacy groups has been central to Prosensa’s mission in identifying and developing effective treatments for DMD,” comments Luc Dochez, Prosensa’s CBO. “The company has already received financial support from Charley’s Fund and other groups during its early years. This additional support for the exon 52 program will enable us to accelerate our efforts toward clinical development.”

Prosensa has two clinical-stage and four preclinical candidates for DMD. Lead compound, PRO051, entered Phase III this January. In 2009, GlaxoSmithKline signed on to help Prosensa develop this drug. GSK also has an option to license Prosensa’s second lead compound, PRO044, which targets the skipping of exon 44. At the time of entering this deal, GSK paid £16 million up front and agreed to pay £412 million in milestones.

In June 2010, the firms expanded their DMD collaboration even further with the initiation of two additional RNA-based drug programs. The two new programs center on the potential development of four candidates that skip different DMD exons: 45, 52, 53, and 55. Under terms of the collaboration, GSK has an option to select two of these additional four compounds for later-stage development and commercialization. Prosensa retains certain limited European commercialization rights alongside GSK for the two compounds selected by GSK. For the two compounds not selected by GSK, Prosensa will retain full commercialization rights.

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