Trial Profile
A Clinical Study Evaluating 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 DD
Status:
Active, no longer recruiting
Phase of Trial:
Phase I
Latest Information Update: 05 Jun 2023
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
- 31 May 2023 According to a Rocket Pharmaceuticals media release, based on data from this study the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to RP-A501.
- 19 May 2023 Results presented in a Rocket Pharmaceuticals media release.
- 19 May 2023 According to a Rocket Pharmaceuticals media release, positive, previously disclosed data (cut-off July 11, 2022) at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles, California.