Rocket Pharmaceuticals

NodThera has appointed Elisabeth Björk, M.D., Ph.D., former Senior Vice President at AstraZeneca's obesity franchise, to its Board of Directors.

The FDA placed a clinical hold on Rocket Pharmaceuticals' Phase 2 pivotal trial for RP-A501, a gene therapy for Danon disease, following a patient death from a serious adverse event.

The FDA has placed a clinical hold on Rocket Pharmaceuticals' Phase II gene therapy trial for RP-A501 after a patient developed capillary leak syndrome and subsequently died.

• REGENXBIO has closed a non-dilutive royalty bond agreement with Healthcare Royalty for up to $250 million, receiving $150 million upfront that extends its cash runway into early 2027. • The deal monetizes select royalties from ZOLGENSMA for SMA and payments from gene therapies for MPS disorders, while REGENXBIO retains other funding opportunities including a potential Priority Review Voucher. • This strategic financing supports REGENXBIO's late-stage pipeline development, including RGX-121 for MPS II, RGX-202 for Duchenne muscular dystrophy, and ABBV-RGX-314 for wet AMD.

Rocket Pharmaceuticals will present preliminary data from its Phase 1 trial of RP-A601 for plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM) at the upcoming ASGCT Annual Meeting on May 15, 2025.

Rocket Pharmaceuticals continues its Phase 2 pivotal study of RP-A501 for Danon disease, with a program update expected mid-year 2025 and clinical data readout anticipated by mid-2026.

An investigational gene therapy has successfully restored immune function in all nine children treated for severe leukocyte adhesion deficiency-I (LAD-I) in an international clinical trial co-led by UCLA.

• Four-year-old Eisa Hussain, born with severe leukocyte adhesion deficiency 1 (LAD-1), is now living a normal life after receiving innovative gene therapy at Great Ormond Street Hospital. • The therapy, which modifies the patient's own cells to create missing proteins needed for immune function, has allowed Eisa to fight infections independently and attend school—outcomes his family never thought possible. • Results from the international clinical trial, published in the New England Journal of Medicine, showed all nine participating children with severe LAD-1 survived to the two-year follow-up mark with sustained clinical benefits.

Rocket Pharmaceuticals' RP-A501 gene therapy demonstrated significant efficacy in Phase 1 trial for Danon Disease, achieving 50% above-normal LAMP2B protein expression in treated patients.

• The Pompe disease pipeline is robust, featuring over 20 drugs in development across 15+ companies, targeting novel treatment approaches. • GeneCradle Therapeutics' GC301, a gene therapy, is in Phase I/II trials, showing potential for enzyme replacement therapy discontinuation and motor ability improvement. • Maze Therapeutics' MZE001, an oral glycogen synthase inhibitor, is in Phase I, aiming to reduce glycogen buildup in Pompe disease patients. • Key players like Amicus Therapeutics and Spark Therapeutics are advancing therapies such as Cipaglucosidase alfa and SPK-3006 through clinical trials.