BioMarin Strikes Gene Therapy Partnership Targeting Rare Genetic Cardiomyopathies

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is beefing up its gene therapy portfolio. The large-cap biopharma BioMarin said it has entered into a pre-clinical collaboration and licensing agreement with DiNAQOR, a gene therapy platform company headquartered in Switzerland, to develop gene therapies to treat rare genetic cardiomyopathies.The licensing agreement will initially cover DiNAQOR’s lead candidate DiNA-001, which is being evaluated for MYBPC3 hypertrophic cardiomyopathy, or HCM.HCM is one of the most common genetic heart diseases, with about 500,000 patients diagnosed, and up to 60% of HCM cases have a genetic origin, BioMarin said.It is estimated that 40% of those have mutations in MYBPC3, the gene that encodes cardiac myosin-binding protein C, the company said. HCM affects the heart muscle, often increasing the risk of heart failure and life-threatening arrhythmias, and there is no currently available pharmacologic treatment option for the disease.BioMarin, which itself is speculated to be an M&A target, said it has the option to extend the license to include DiNAQOR’s other pipeline programs on similar terms.BioMarin said it is also picking up a stake in DiNAQOR.The financial terms of the deal were not disclosed.BioMarin also reiterated its 2020 GAAP net income guidance of $20 million to $80 million, after factoring in this collaboration.”With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology,” BioMarin CEO Jean-Jacques Bienaime said in a statement. BioMarin’s BLA for AAV5 gene therapy candidate valoctocogene roxaparvovec has been accepted for review by the FDA with a priority review designation. The gene therapy, which is being evaluated for a bleeding disorder, has a PDUFA action date of Aug. 21.BioMarin shares were down 1.44% at $86.85 at the time of publication Monday. Related Links:Attention Biotech Investors: Mark Your Calendar For These May PDUFA Dates The Week Ahead In Biotech: Smid-Cap Earnings News Flow Picks Up Pace See more from Benzinga * The Daily Biotech Pulse: Inovio’s MERS Vaccine Data, Fast Track Designation For Erytech, FDA Approves Higher Dose Of Merck’s Keytruda * The Daily Biotech Pulse: Pfizer Earnings, Roche’s Spinal Muscular Dystrophy Treatment, Moderna COVID-19 Vaccine Update * The Week Ahead In Biotech (April 26-May 2): Cancer Conference, Earnings News Flow In The Spotlight(C) 2020 Benzinga does not provide investment advice. All rights reserved.