Gene Therapy for Danon Disease (DD): A Clinical Study Evaluating the Infusion of a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease
Phase of Trial: Phase I
Latest Information Update: 05 Dec 2019
Price : $35 *
At a glance
- Drugs RP A501 (Primary)
- Indications Glycogen storage disease type II
- Focus Adverse reactions; First in man; Pharmacodynamics; Pharmacogenomic; Therapeutic Use
- Sponsors Rocket Pharmaceuticals
- 07 Aug 2019 According to a Rocket Pharmaceuticals media release, patient dosing continues in first cohort.
- 18 Jun 2019 According to a Rocket Pharmaceuticals media release, patient dosing has commenced in this trial.
- 08 May 2019 According to a Rocket Pharmaceuticals media release, data from this trial is expected in 2020.