SPY322.64-7.66 -2.32%
DIA267.58-5.23 -1.92%
IXIC10,632.99-330.65 -3.02%

Rocket Pharmaceuticals Expands Clinical Sites for its FA, Danon, LAD-I and IMO Trials

—Four Clinical Trial Sites Added Across Multiple Clinical Programs, Including Great Ormond Street Hospital, Children’s Hospital of Philadelphia, University of Minnesota and University of California, Los

Benzinga · 08/26/2020 11:01

—Four Clinical Trial Sites Added Across Multiple Clinical Programs, Including Great Ormond Street Hospital, Children’s Hospital of Philadelphia, University of Minnesota and University of California, Los Angeles—

—New Sites Expand Opportunities for Patient Access, Enrollment and Overall Presence Globally—

NEW YORK--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the addition of four additional clinical trial sites across four clinical programs for Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Danon Disease and Infantile Malignant Osteopetrosis (IMO). The newly added centers, which include some of the leading gene therapy research programs in the world, are: Great Ormond Street Hospital (GOSH), Children’s Hospital of Philadelphia (CHOP), the University of Minnesota and the University of California, Los Angeles (UCLA).

“Rocket is pleased to add these world-class sites which represents another meaningful step in our journey and vision of having Centers of Excellence worldwide,” said Kinnari Patel, Pharm.D., MBA, Chief Operating Officer and Head of Development of Rocket. “We have been working with prominent and experienced research centers to broaden our footprint to new geographies for these trials and the entire pipeline overall. These sites represent not only world-class partners for expanding patient enrollment but ideal partners for any future potential commercialization. We are confident that the exceptional principal investigators at each of these sites will strengthen our already robust clinical trial activities and expand access for patients contending with rare and devastating disorders.”

Great Ormond Street Hospital in London has opened as a clinical trial site for Rocket’s FA and LAD-I programs. Dr. Claire Booth, Mahboubian Associate Professor in Gene Therapy University College London Great Ormond Street Institute of Child Health (UCL GOS ICH) and Honorary Consultant in Pediatric Immunology, GOSH, will serve as principal investigator at the site. GOSH, together with its academic partner the UCL GOS ICH forms the largest concentration of children’s health research in Europe. The LAD-I trial first opened at the University of California, Los Angeles, and two patients have already been treated in the Phase 1 trial.

As Rocket moves into the next cohort in its clinical trial of RP-A501 for the treatment of Danon Disease, the world-renowned Children’s Hospital of Philadelphia will join the University of California San Diego (UCSD) Health as a trial site under the leadership of Joseph Rossano, M.D., M.S., FAAP, FACC, Chief of the Division of Cardiology at CHOP and Professor of Pediatrics at Perelman School of Medicine at the University of Pennsylvania. With this addition, Rocket brings an East Coast presence to the trial, facilitating broader patient access.

Rocket’s global registrational trial of RP-L102 for FA continues to progress and will expand to the University of Minnesota under the leadership of Margaret MacMillan, M.D., MSc., FRCPC, Professor in Pediatrics in the Division of Blood and Marrow Transplantation at the University of Minnesota Medical School.

In addition, the trial site for the recently launched Phase 1 clinical trial of RP-L401 for IMO will be University of California, Los Angeles led by principal investigator Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los Angeles and principal investigator of RP-L201 for LAD-I. The presence of UCLA and addition of Dr. Kohn are major steps forward in Rocket’s continued evaluation of RP-L401 as a treatment for IMO.

The collaborations with GOSH/ICH and University of Minnesota further leverage the ongoing consultative expertise of Adrian Thrasher, Ph.D., M.D. and John Wagner, M.D., respectively. Professor Thrasher is Professor of Pediatric Immunology and Wellcome Trust Principal Research Fellow at the ICH. Dr. Wagner is Director of the Institute of Cell, Gene and Immunotherapeutics at the University of Minnesota. Since 2016, Professor Thrasher has collaborated with Rocket regarding multiple lentiviral programs and lends valuable insight through his role at the largest concentration of children’s health research in Europe. Dr. Wagner is an internationally recognized expert in FA, stem cell and transplantation biology, and umbilical cord transplant. Dr. Wagner has been closely involved in Rocket’s FA program since 2018.