美国食品药品监督管理局对心脏病项目实施临床搁置，损害我们的核心论点

Andrew Tsai * | Equity Analyst(415) 229-1566 | atsai@jefferies.comMatthew Barcus, Ph.D. * | Equity Associate(415) 229-8703 | mbarcus@jefferies.comJohn Cox, Ph.D. * | Equity Associate+1 (415) 229-8708 | jcox1@jefferies.comBrian Balchin, ACA * | Equity Analyst(415) 229-1522 | bbalchin@jefferies.com the immunosuppressive regimen via earlier steroid taper (sirolimusinstead oftacrolimus)andemployed more rigorous early monitoring of complement activation.Please see important disclosure information on pages 3 - 8 of this report.This report is intended for Jefferies clients only. Unauthorized distribution is prohibited. Company DescriptionRocket PharmaceuticalsBased in New Jersey, Rocket Pharmaceuticals (RCKT) is a fully-integrated, late-stage biotechnology company advancing genetic medicines for thetreatment of rare devastating diseases. RCKT's general strategy revolves around developing novel, disease-modifying genetic medicines for rarediseases with no competition. The company has five programs in clinical development including both ex vivo (cells first modified outside the bodyand then reinserted) and in vivo (new genetic material introduced into the patient) gene therapies, with a sixth program to enter the clinic in 2025.Indeed, two of RCKT's ex vivo gene therapies are in the regulatory filing stages, with RP-L201 (Kresladi) for LAD-I and RP-L102 for Fanconi anemiaboth potentially approved in 2025. For the in vivo gene therapy side, RCKT's lead program RP-A501 is in a pivotal Phase II study for Danon disease,which could have data around YE:25.Company Valuation/RisksRocket PharmaceuticalsOur price target is DCF-based. Risks include clinical, regulatory, and commercial.Analyst Certification:I, Andrew Tsai, certify that all of the views expressed in this research report accurately reflect my personal views about the subject security(ies) and subjectcompany(ies). I also certify that no part of my compensation was, is, or will be, directly or indirectly, related to the specific recommendations or views expressedin this research report.I, Matthew Barcus, Ph.D., certify that all of the views expressed in this research report accurately reflect my personal views about the subject security(ies) andsubject company(ies). I also certify that no part of my compensation was, is, or will be, directly or indirectly, related to the specific recommendations or viewsexpressed in this research report.I, John Cox, Ph.D., certify that all of the views expressed in this research report accurately reflect my personal views about the subject security(ies) and subjectcompany(ies). I also certify that no part of my compensation was, is, or will be, directly or indirectly, related to the specific recommendations or views expressedin this research report.I, Brian Balchin, ACA, certify that all of the views expressed in this research report accurately reflect my personal views about the subject security(ies) and subjectcompany(ies). I also certify that no part of my compensation was, is, or will be, directly or indirectly, related to the specific recommendations or views expressedin this research report.As is the case with all Jefferies employees, the analyst(s) responsible for the coverage of the financial instruments discussed in this report receives compensationbased in part on the overall performance of the firm, including investment banking income. We seek to update our research as appropriate, but various regulationsmay prevent us from doing so. Aside from certain industry reports published on a periodic basis, the large majority of reports are published at irregular intervalsas appropriate in the analyst's judgement.Investment Recommendation Record(Article 3(1)e and Article 7 of MAR)Recommendation PublishedRecommendation DistributedExplanation of Jefferies RatingsBuy - Describes securities that we expect to provide a total return (price appreciation plus yield) of 15% or more within a 12-month period.Hold - Describes securities that we expect to provide a total return (price appreciation plus yield) of plus 15% or minus 10% within a 12-month period.Underperform - Describes securities that we expect to provide a total return (price appreciation plus yield) of minus 10% or less within a 12-month period.The expected total return (price appreciation plus yield) for Buy rated securities with an average security price consistently below $10 is 20% or more within a 12-month period as these companies are typically more volatile than the overall stock market. For Hold rated securities with an average security price consistentlybelow $10, the expected total return (price appreciation plus yield) is plus or minus 20% within a 12-month period. For Underperform rated securities with anaverage security price consistently below $10, the expected total return (price appreciation plus yield) is minus 20% or less within a 12-month period.NR - The investment rating and price target have been temporarily suspended. Such suspensions are in compliance