You should read the following discussion and analysis of our financial conditionand results of operations together with the consolidated financial statementsand related notes that are included elsewhere in this Quarterly Report on Form10-Q and our 2021 Form 10-K. This discussion contains forward-looking statementsbased upon current plans, expectations and beliefs that involve risks anduncertainties. Our actual results may differ materially from those anticipatedin these forward-looking statements as a result of various factors, including,but not limited to, those discussed in the section entitled "Risk Factors" andelsewhere in this Quarterly Report on Form 10-Q. In preparing this MD&A, wepresume that readers have access to and have read the MD&A in our 2021 Form10-K, pursuant to Instruction 2 to paragraph of Item 303 of Regulation S-K.Unless stated otherwise, references in this Quarterly Report on Form 10-Q to"us," "we," "our," or our "Company" and similar terms refer to RocketPharmaceuticals, Inc.We are a clinical-stage, multi-platform biotechnology company focused on thedevelopment of first, only and best-in-class gene therapies, with directon-target mechanism of action and clear clinical endpoints, for rare anddevastating diseases. We have three clinical-stage ex vivo lentiviral vector("LVV") programs. These include programs for Fanconi Anemia ("FA"), a geneticdefect in the bone marrow that reduces production of blood cells or promotes theproduction of faulty blood cells, Leukocyte Adhesion Deficiency-I ("LAD-I"), agenetic disorder that causes the immune system to malfunction and PyruvateKinase Deficiency ("PKD"), a rare red blood cell autosomal recessive disorderthat results in chronic non-spherocytic hemolytic anemia. Of these, both thePhase 2 FA program and the Phase 1/2 LAD-I program are in potentiallyregistration-enabling studies in the United States ("U.S.") and Europe ("EU").In addition, in the U.S., we have a clinical stage in vivo adeno-associatedvirus ("AAV") program for Danon disease, a multi-organ lysosomal-associateddisorder leading to early death due to heart failure. Additional work on a genetherapy program for the less common FA subtypes C and G is ongoing. We haveglobal commercialization and development rights to all of these productcandidates under royalty-bearing license agreements.Effective December 2021, a decision was made to no longer pursueRocket-sponsored clinical evaluation of RP-L401; this program was returned toacademic innovators. Although we believe that gene therapy may be beneficial topatients afflicted with this disorder, we have opted to focus availableresources towards advancement of RP-A501, RP-L102, RP-L201 and RP-L301, based onthe compelling clinical data to date and potential for therapeutic advancementin these severe disorders of childhood and young adulthood.

Recent Developments

At-the-Market Offering Program

Gene Therapy Overview

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Index

The chart below shows the current phases of development of Rocket's programs andproduct candidates:

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Analysis of the explanted heart revealed significant fibrosis consistent with

advanced DD.

Myocardial tissue from the explanted heart at 5 months post-treatment displayed

100% LAMP2B protein expression by immunohistochemistry throughout non-fibrotic

cardiac regions including the ventricles and other essential targeted areas

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Fanconi Anemia Complementation Group A (FANCA):

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Leukocyte Adhesion Deficiency-I (LAD-I):

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Pyruvate Kinase Deficiency (PKD):

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Infantile Malignant Osteopetrosis (IMO):

cGMP Manufacturing

Strategy

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Financial Overview

Revenue

Operating Expenses

Research and Development Expenses

Our R&D program expenses consist primarily of external costs incurred for thedevelopment of our product candidates. These expenses include:

expenses incurred under agreements with research institutions and consultants

that conduct R&D activities including process development, preclinical, and

clinical activities on our behalf;

costs related to process development, production of preclinical and clinical

materials, including fees paid to contract manufacturers and manufacturing

input costs for use in internal manufacturing processes;

consultants supporting process development and regulatory activities;

patent fees; and

costs related to in-licensing of rights to develop and commercialize our

product candidate portfolio.

salaries and personnel-related costs, including benefits, travel, and

stock-based compensation, for our scientific personnel performing R&D

activities;

facilities and other expenses, which include expenses for rent and maintenance

of facilities, and depreciation expense; and

laboratory supplies and equipment used for internal R&D activities.

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$ 4,507 $ 15,942 $ 8,307Leukocyte Adhesion Deficiency (LVV) RP-L201

$ 5,078 $ 12,511 $ 9,741Stock based compensation expense

$ 24,530 $ 72,150 $ 52,839

(1) Effective December 2021, a decision was made to no longer pursue

Rocket-sponsored clinical evaluation of RP-L401; this program was returned to

the scope, rate of progress, and expense of ongoing as well as any clinical

studies and other R&D activities that we undertake;

future clinical study results;

uncertainties in clinical study enrollment rates;

changing standards for regulatory approval; and

the timing and receipt of any regulatory approvals.

the scope, progress, outcome and costs of our clinical trials and other R&D

activities;

the efficacy and potential advantages of our product candidates compared to

alternative treatments, including any standard of care;

the market acceptance of our product candidates;

obtaining, maintaining, defending, and enforcing patent claims and other

intellectual property rights;

significant and changing government regulation; and

the timing, receipt, and terms of any marketing approvals.

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General and Administrative Expenses

Interest Expense

Interest Income

Interest income is related to interest earned from investments and cashequivalents.

Critical Accounting Policies and Significant Judgments and Estimates

Comparison of the Three Months Ended June 30, 2022 and 2021

-

168

Amortization of premium on investments - net (396 ) (727 )

Research and Development Expenses

General and Administrative Expenses

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Comparison of the Six Months Ended June 30, 2022 and 2021

1,052

Research and Development Expenses

General and Administrative Expenses

Other Expense, Net

Liquidity, Capital Resources and Plan of Operations

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Index

20,178

9,907

Net change in cash, cash equivalents and restricted cash $ (45,146 )

Investing Activities

During the six months ended June 30, 2022, net cash provided by investingactivities was $15.8 million, primarily resulting from proceeds of $163.7million from the maturities of investments, offset by purchases of investmentsof $143.0 million, and purchases of property and equipment of $4.8 million.

Financing Activities

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ROCKET PHARMACEUTICALS, INC. Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) - Marketscreener.com

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