RP-L401 / Rocket Pharma - LARVOL DELTA

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis (clinicaltrials.gov) - P1 | N=1 | Terminated | Sponsor: Rocket Pharmaceuticals Inc. | Active, not recruiting ➔ Terminated; Study discontinued due to feasibility.

Trial termination • Gene Therapies

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis (clinicaltrials.gov) - P1; N=2; Active, not recruiting; Sponsor: Rocket Pharmaceuticals Inc.; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Gene Therapies

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis (clinicaltrials.gov) - P1; N=2; Recruiting; Sponsor: Rocket Pharmaceuticals Inc.; Not yet recruiting ➔ Recruiting; Initiation date: Oct 2020 ➔ Feb 2021

Clinical • Enrollment open • Trial initiation date • Gene Therapies

"#RocketPharmaceuticals Receives #FDA #FastTrackDesignation for #RPL401 #GeneTherapy for #InfantileMalignantOsteopetrosis $RCKT https://t.co/DlqmVlY2G9" (@1stOncology)

Fast track designation • Gene Therapies

A Trial to Evaluate Safety and Efficacy of Autologous CD34+ Cells With a Lentiviral Vector Encoding the TCIRG1 Gene (clinicaltrials.gov) - P1; N=2; Not yet recruiting; Sponsor: Rocket Pharmaceuticals Inc.

New P1 trial • Gene Therapies