Rocket Pharmaceuticals is laying off 30% of its staff and focusing on its cardiovascular disease programs, causing delays to assets including a gene therapy that was on track to win approval next year.
The biotech suffered a setback in May when a participant in its pivotal Danon disease gene therapy trial died and the FDA put a clinical hold on the study. Rocket responded by prioritizing investments in its adeno-associated virus platform and starting a strategic review to extend its cash runway into 2027, compared to late 2026 under the previous plan. The biotech fleshed out its response Thursday.
Rocket, which ended last year with 299 full-time employees, is working to reduce its head count by 30%. The company began notifying affected employees on Wednesday and is aiming to complete the layoffs by Aug. 8. Rocket expects to incur costs of $3.5 million, mainly from severance payments to 80 people.
The company is making the layoffs in conjunction with a narrowing of its focus. Rocket’s pipeline is split between cardiovascular and hematology assets. Going forward, Rocket will focus on the cardiovascular side of the pipeline, namely the Danon program and treatments for two forms of cardiomyopathy.
Leukocyte adhesion deficiency-I gene therapy Kresladi is the only hematology candidate on Rocket’s list of priority programs. The FDA rejected a request for approval of the asset last year, citing the need for additional manufacturing information. Rocket has earmarked resources for submitting responses to the complete response letter.
The company could bag a priority review voucher if Kresladi wins approval. Rocket hasn’t factored the PRV into its cash runway, which now extends into the second quarter of 2027, but the voucher could be lucrative for the company. PRVs have been selling for $150 million or more.
Rocket has extended its cash runway by diverting resources away from two hematology programs. The company was aiming to win FDA approval for RP-L102 in Fanconi anemia next year but has axed that goal as part of the pipeline prioritization. Rocket said it also expects the changes to delay its pyruvate kinase deficiency program RP-L301.