Rocket Pharmaceuticals Announces Clearance of IND for RP-L201 Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I)

- Phase 1/2 Clinical Trial in Support of Registration to Commence in the U.S. in 2019 -
- Trial to Assess Safety, Tolerability and Efficacy of RP-L201 -

NEW YORK, NY, USA I November19, 2018 I Rocket Pharmaceuticals, Inc. (Nasdaq:RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, today announces the clearance of the Company’s Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for RP-L201. RP-L201 is the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I) that was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT).

“This acceptance marks the second Rocket-Sponsored IND cleared for our ex-vivo lenti platform this quarter. We are excited to achieve this important milestone ahead of schedule, and look forward to working with the medical and patient communities to evaluate RP-L201 next year,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “This IND enables Rocket to initiate a Phase 1/2 clinical trial to assess the safety, tolerability, and efficacy of severe LAD-I in children. Severe LAD-I is a devastating pediatric disease and we believe that we may have an opportunity to significantly improve the lives of very young children suffering from this disease with a potentially curative therapy.”

The planned open-label, single-arm, Phase 1 portion of the clinical trial of RP-L201 is expected to enroll two severe LAD-I patients in the U.S. The Phase 2 portion of the clinical trial of RP-L201 is expected to be a registration-enabling global study with clinical sites in the U.S., U.K. and Spain.

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, life-threatening, autosomal recessive pediatric disease caused by a mutation of the ITGB2 gene that encodes for the Beta-2 Integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and enables neutrophil extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is exceedingly rare. The unmet medical need for patients with severe LAD-I is therefore significant.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rocket’s multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's lead clinical program is a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. Preclinical studies of additional bone marrow-derived disorders are ongoing and target Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO). Rocket is also developing an AAV-based gene therapy program for an undisclosed rare pediatric disease. For more information about Rocket, please visit

SOURCE: Rocket Pharmaceuticals

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