Rocket Pharmaceuticals to acquire Renovacor

By Rachel Arthur

- Last updated on GMT


Related tags Heart disease AAV

Rocket Pharmaceuticals, a late-stage, clinical biotech advancing a pipeline of genetic therapies for rare childhood disorders, will acquire Renovacor: helping it strengthen its position in AAV-based cardiac gene therapy.

The $53m deal will create ‘strong synergies’ by combining key assets, personnel, capabilities and IP; while Rocket gains Renovacor’s REN-001, an AAV-based gene therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a severe form of heart failure (for which Rocket highlights ‘compelling preclinical data’ generated by Renovacor)

New Jersey headquartered Rocket currently has four gene therapies in clinical trials. This includes a Phase 1 trial for rare genetic disorder Danon Disease; and it sees a 'comparable, meaningful commercial opportunity' for BAG3-associated dilated cardiomyopathy. 

“The acquisition of Renovacor aligns with our strategy to expand our leadership position in AAV-based gene therapy for cardiac disease and gives us a perfect opportunity to continue on our mission to transform the lives of heart failure patients through the power of gene therapy,”​ said Gaurav Shah, M.D., CEO of Rocket.

“Building on our success in Danon Disease to date, I am particularly excited to expand our cardiology focus and capabilities and address a clear unmet medical need in BAG3-associated dilated cardiomyopathy. By combining Renovacor’s compelling preclinical work with our joint clinical, regulatory and CMC expertise, we believe we will be well-positioned to bring the highest impact gene therapy with the best chance for success to these patients in the most productive and efficient manner possible.

“Given the positive pediatric safety data previously announced from our Phase 1 RP-A501 Danon Disease program, and the upcoming pediatric efficacy data and longer-term adult cohort data we anticipate presenting at the Heart Failure Society of America (HFSA) Scientific Meeting at the end of this month, this strategic acquisition gives us what we believe is the broadest platform in the field to address these devastating rare cardiac diseases.

"Furthermore, the acquisition will bring to Rocket key personnel, namely a team of leading cardiology drug development experts, critical capabilities, and valuable IP to support continued development of the BAG3 as well as other potential cardiac programs, including a gene therapy research collaboration for arrhythmogenic cardiomyopathy.”

Massachusetts-headquartered Renovacor’s most advanced program, REN-001, is an AAV-based gene therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a severe form of heart failure (BAG3-DCM represents a ‘significant unmet medical need’ in a patient population with rapidly progressive cardiac dysfunction in whom no treatments targeting the underlying mechanism of disease exist). Renovacor has designed its gene replacement therapy to treat BAG3-associated DCM through in vivo delivery of a human BAG3 gene to cardiomyocytes.

The transaction is expected to close by the first quarter of 2023, subject to approval by Renovacor and Rocket shareholders, receipt of any required customary regulatory approvals and the satisfaction of other customary closing conditions.

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