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Rocket Pharmaceuticals NASDAQ:RCKT Rocket Pharmaceuticals, Inc. is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Company's platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia, and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rocket's first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.

Location: 350 5th Ave Ste 7530, New York, 10118-7501, US | Website: www.rocketpharma.com | Industry: Pharmaceutical Preparation Manufacturing | Sector: Manufacturing

Market Cap

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52 Wk Range

$ - $

Previous Close

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Open

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Day Range

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Volume

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Avg Volume

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Enterprise Value

328.1M

Cash

372.3M

Avg Qtr Burn

-52.43M

Short % of Float

15.40%

Insider Ownership

3.02%

Institutional Own.

Qtr Updated

12/31/24

Drug Pipeline

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Drug & Indication	Stage & Event	Catalyst Date
KRESLADI (Marnetegragene autotemcel) /RP-L201 Details Immunodeficiency, Leukocyte Adhesion Deficiency	BLA Resubmission	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
RP-L102 Details Rare diseases, Blood disorder, Fanconi Anemia	BLA Submission	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
RP-A501 Details Immunodeficiency, Danon Disease	Phase 2 Update	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
RP-L301 Details Pyruvate Kinase Deficiency	Phase 2 Initiation	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
RP-A601 Details Heart disease, Arrhythmogenic cardiomyopathy	Phase 1 Data readout	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
RP-L401 Details Infantile Malignant Osteopetrosis	Failed Discontinued	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.