DMD Pipeline: After Sarepta’s First-Ever Approval, Are Combinations Next?
Several companies have DMD drug candidates waiting in the wings to follow Sarepta’s Exondys 51 into the commercial market after FDA approval of the exon-skipping therapy, which could be the backbone for future combination treatment regimens.
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Although only interim data from six treated patients, Capricor’s Phase II DMD study shows improvements in upper limb performance, grip strength and respiration. Cardiac performance shows positive trend, but not yet statistical significance.
Gossamer Bio, led by former Receptos CEO Faheem Hasnain and CMO Sheila Gujrathi, revealed $100m in seed and Series A funding during a week in which 17 companies revealed VC rounds totaling $891.2m, giving them good news to discuss during the J.P. Morgan Healthcare conference.
California biotech says it will move forward with knowledge and expertise gained through the failed partnership. Capricor hopes to re-partner CAP-1002 for cardiovascular indications, while continuing the candidate’s DMD development itself.