Rocket Pharmaceuticals to Host In-Person Investor and Analyst Event and Webcast on Tuesday, Dec. 14 at 7:30 a.m. ET to Discuss Updated Clinical Data Presented at the 63rd American Society of Hematology (ASH) Annual Meeting

Rhea-AI Summary

Rocket Pharmaceuticals (NASDAQ: RCKT) will host an Investor and Analyst Event on December 14, 2021 at 7:30 a.m. ET in Atlanta, featuring updates on its gene therapy programs targeting Leukocyte Adhesion Deficiency-I (LAD-I), Fanconi Anemia (FA), and Pyruvate Kinase Deficiency (PKD). Clinical data from these Phase 1 and Phase 2 trials will also be shared at the American Society of Hematology Annual Meeting (Dec. 11-14, 2021). The company is focused on developing therapies for rare childhood disorders through its innovative lentiviral vector (LVV) approach.

Positive

• Hosting an investor event to provide clinical updates on key gene therapy programs.
• Presentation of data at a prestigious platform like the American Society of Hematology Annual Meeting.
• Focus on treating serious conditions like LAD-I, FA, and PKD, indicating strong research and development commitment.

Negative

• None.

—Data updates to be presented from ongoing Phase 2 registrational trials in LAD-I and FA and Phase 1 trial in PKD —

CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, will host an in-person Investor and Analyst Event in Atlanta that will simultaneously be webcast on Tuesday, Dec. 14, 2021, at 7:30 a.m. ET to discuss updates from three of the Company’s lentiviral vector (LVV)-based gene therapy programs: RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I), RP-L102 for Fanconi Anemia (FA) and RP-L301 for Pyruvate Kinase Deficiency (PKD). Clinical data for all three trials will be presented at the 63^rd American Society of Hematology (ASH) Annual Meeting being held Dec. 11-14, 2021.

Investors may register to attend the event in person by emailing investors@rocketpharma.com. A simultaneous webcast of the event and the presentation will be available under “Events” in the Investors section of the Company’s website at: https://ir.rocketpharma.com/.

The webcast replay will be available on the Rocket website upon completion of the event. This meeting is NOT an official program of the ASH annual meeting.

About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) 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. For more information about Rocket, please visit www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements
Various statements in this release concerning Rocket’s future expectations, plans and prospects, including without limitation, Rocket’s expectations regarding its guidance for 2021 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, Rocket’s plans for the advancement of its Danon Disease program following the lifting of the FDA’s clinical hold and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2020, filed March 1, 2021 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211207005330/en/

Media

Kevin Giordano

Director, Corporate Communications

kgiordano@rocketpharma.com

Investors

Mayur Kasetty, M.D., M.B.A.

Director, Business Development & Operations

investors@rocketpharma.com

Source: Rocket Pharmaceuticals, Inc.

FAQ

What will Rocket Pharmaceuticals present at the December 14, 2021 event?

Rocket Pharmaceuticals will present updates on its gene therapy programs for LAD-I, FA, and PKD, including clinical data from ongoing trials.

When is the American Society of Hematology Annual Meeting?

The American Society of Hematology Annual Meeting is scheduled for December 11-14, 2021.

How can investors attend the event hosted by Rocket Pharmaceuticals?

Investors can register to attend in person by emailing investors@rocketpharma.com.

What is the significance of the clinical data presentation?

The presentation of clinical data at a major conference highlights the progress and potential impact of Rocket's therapies for rare genetic disorders.