Rocket Pharma ((RCKT)) announced an update on their ongoing clinical study.
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Rocket Pharmaceuticals is conducting a Phase 2 clinical study titled ‘Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease.’ The study aims to assess the efficacy and safety of RP-A501, a gene therapy product, in treating male patients with Danon Disease, a rare genetic disorder.
The intervention being tested is RP-A501, a genetic therapy involving a recombinant adeno-associated virus serotype 9 (AAV9) vector carrying the human LAMP2B transgene. It is administered as a single intravenous infusion to target the underlying genetic cause of Danon Disease.
This interventional study follows a single-group assignment model without masking, focusing on treatment as its primary purpose. Participants receive one planned dose of RP-A501, and the study does not involve any control groups.
The study began on October 11, 2023, and is currently recruiting participants. The primary completion and estimated study completion dates are yet to be announced. The last update was submitted on October 1, 2025, indicating ongoing progress.
This update could influence Rocket Pharma’s stock performance positively, as successful results may enhance investor confidence and position the company as a leader in gene therapy for rare diseases. Investors should also consider the competitive landscape, as advancements in gene therapy are closely watched by industry peers.
The study is ongoing, with further details available on the ClinicalTrials portal.