NASDAQ Framework: Rocket Pharmaceuticals Inc.
Late-stage biotechnology company developing gene therapies 1.
1: Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey. View Source
Rocket Pharmaceuticals, Inc., headquartered in Cranbury, New Jersey, is a fully integrated, late-stage biotechnology company focused on developing gene therapies for rare and devastating diseases 23. The company is advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders, with clinical-stage programs targeting hematologic diseases such as Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency, as well as in vivo adeno-associated virus programs for conditions like Danon disease and Plakophilin-2 Arrhythmogenic Cardiomyopathy 4. Rocket Pharmaceuticals is committed to making a lasting impact on patients' lives through innovative treatments and strong community engagement 56.
2: Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey. View Source3: Rocket Pharmaceuticals, Inc. is a fully integrated, late-stage biotechnology company. The Company is advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Its lentiviral (LV) vector-based gene therapies target hematologic diseases and consist of late-stage programs for Fanconi Anemia, a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte... View Source4: Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey. View Source5: Rocket Pharmaceuticals 27,125 followers 2d Report this post #TeamRocket would like to extend our heartfelt gratitude to Barry Greenberg, Distinguished Professor of Medicine, Director, Advance Heart Failure Treatment Program UC San Diego, and all the presenters who contributed to the success of the 30th Annual San Diego Heart Failure Symposium! For 30 years, this flagship event organized by Dr. Greenburg has brought together the scientific and medical communities to explore the latest advances in the diagnosis, research, and treatment of heart failure. We are honored to again sponsor this conference and engage with the heart failure community as we seek #GeneTherapy cures for patients with rare cardiovascular diseases. Learn more: www.rocketpharma.com. #SDHF2024 44 Like Comment Share Rocket Pharmaceuticals 27,125 followers 5d Report this post #TeamRocket is thrilled to sponsor the 30th Annual San Diego Heart Failure Symposium. Engaging with the medical and scientific communities at this annual event is invaluable in driving forward advances in the recognition and treatment of heart failure. Our team of innovators are hard at work advancing our #AAV #GeneTherapy cardiovascular pipeline of potentially curative treatment options for patients with high unmet needs. Learn more: https://lnkd.in/evPEDXPk. #SDHF2024 31 Like Comment Share Rocket Pharmaceuticals 27,125 followers 1w Report this post #TeamRocket had the honor of engaging with the #PKD community at #BridgingLivesPKD, the 2nd Annual International PK Deficiency Conference hosted by Thrive with Pyruvate Kinase Deficiency and the Pyruvate Kinase Deficiency Foundation. Patients are our North Star, and having meaningful interactions with patients and advocates fuels our team’s pursuit of #GeneTherapy cures. Thanks to everyone who made this event a success and for your unwavering support for the PKD patient and advocacy community. #PKDConference #PKDeficiency 30 Like Comment Share Rocket Pharmaceuticals 27,125 followers 1w Report this post #TeamRocket recently came together to honor our commitment to the community during our annual Rocket Day of Service, celebrated on the day of Rocket’s founding. From Cranbury, NJ, to NYC and beyond, our employees volunteered their time and energy to make a positive impact on those in need. Our mission extends beyond #GeneTherapy; it’s about serving people. At Rocket, our Day of Service embodies this spirit by reinforcing Generosity as one of our core values, strengthening our bond as a team, and celebrating our company's founding by making a positive impact. Thank you to all the members of our team for their generosity and dedication to serving others. Whether participating in on-site activities, local projects, or individual volunteer efforts in the community, each member played a vital role in making this day a success. 🌍❤️🚀 #RocketPharma #DayOfService #RocketDayofService 121 2 Comments Like Comment Share Rocket Pharmaceuticals 27,125 followers 1w Report this post Patients are our North Star and inspiration. We are honored to once again sponsor the #BridgingLivesPKD Conference in partnership with the Pyruvate Kinase Deficiency Foundation and Thrive with Pyruvate Kinase Deficiency. #TeamRocket is advancing an investigational #GeneTherapy program and committed to our goal of making a lasting impact on the lives of patients and families living with #PKDeficiency. Learn more: https://thrivewithpkd.org/. #PKDConference #PyruvateKinaseDeficiency #PKD 46 Like Comment Share Rocket Pharmaceuticals 27,125 followers 3w Report this post #TeamRocket is thrilled to welcome Isabel Cheng, PharmD & Sonali Limbachia, PharmD from the Rutgers Pharmaceutical Industry Fellowship (RPIF) Program! Participating in this fellowship program underscores our commitment to fostering innovation, nurturing talent, and shaping future leaders who will continue our mission for years to come. Join us in expressing appreciation for Sanchali Kasbekar, Pharm.D., R.Ph and Ed Trzaska, our preceptors, for investing their time and expertise into this process and their commitment to developing future industry talent. #GeneTherapy #Fellowship #WelcomeAboard 149 4 Comments Like Comment Share Rocket Pharmaceuticals 27,125 followers 4w Edited Report this post #TeamRocket recently celebrated #PrideMonth🌈 at our R&D and manufacturing facility in Cranbury, NJ. We foster a culture of creativity, compassion, and dedication, empowering each employee to be their authentic self. Like puzzle pieces, our diverse strengths come together to create a cohesive team of more than 300 employees dedicated to seeking #GeneTherapy cures for patients with rare and devastating diseases. Learn more about our culture: https://lnkd.in/exrgX9Pj. #TeamCulture #Pride 101 Like Comment Share Rocket Pharmaceuticals 27,125 followers 4w Edited Report this post We’re proud to share that #TeamRocket's Latika Hickey (Pandey), PharmD, Senior Director of Patient Advocacy, has been nominated in the Global Genes #RAREChampionsOfHope awards as a #RARE Champion in Advocacy and Industry! These awards recognize an individual rare disease patient or advocate who has created innovative programming and/or successful partnerships to support, educate, research, or enable their communities in meaningful and impactful ways. Please join us in congratulating Latika and the rest of the 2024 nominees for their dedication to improving the lives of patients and their loved ones impacted by #RareDiseases. Learn more: https://lnkd.in/gHnQeecy. #KnowRare #RareInsightsPodcast 192 33 Comments Like Comment Share Rocket Pharmaceuticals 27,125 followers 1mo Report this post #TeamRocket's President, Head of R&D and COO, Kinnari Patel, PharmD, MBA, joined Taren Grom, Editor and Co-Founder of PharmaVoice, to discuss how we are making a difference for patients and driving innovation in the Know Rare Insights Podcast. Listen to learn how we’re prioritizing patients through the drug development process with the goal of developing first and best-in-class #GeneTherapy treatment options. Listen now: https://lnkd.in/dPbNSfTM. #RareDisease #KnowRare #RareInsightsPodcast …see more #1 Sustaining A Rare Disease Pipeline with Rocket Pharma | Rare Insights: Uncovering The Future Of Rare Disease Treatments rareinsights.podbean.com 72 Like Comment Share Rocket Pharmaceuticals 27,125 followers 1mo Report this post Today, we recognize Juneteenth, a historic day marking the end of slavery in the United States. This day serves as a powerful reminder of the resilience, strength, and enduring spirit of the African American community. Let's take this opportunity to honor the struggles and achievements of those who fought for freedom and equality. It's a time for reflection, education, and action towards a more just and equitable society. #TeamRocket stands in solidarity with the African American community, recognizing the importance of this day and committing to supporting the fight against injustice. #Juneteenth #FreedomDay #BlackHistory 35 Like Comment Share View Source6: Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey. View Source
UNKNOWN
The provided facts do not specify whether Rocket Pharmaceuticals Inc. is still led by its founders or if the founders are heavily involved in the current leadership.
The provided facts do not specify whether Rocket Pharmaceuticals Inc. is still led by its founders or if the founders are heavily involved in the current leadership.
MEDIUM RISK
Rocket Pharmaceuticals Inc. has a mixed financial outlook. While the company is advancing a sustainable pipeline of investigational genetic therapies and has received positive updates such as the Orphan Medicinal Product Designation from the European Commission 7, it also faces significant challenges. The FDA has rejected its gene therapy for a rare immune disorder, asking for more data, which has negatively impacted its stock 8. Additionally, the company has experienced a price target downgrade from multiple financial institutions 9. These factors indicate that while Rocket Pharmaceuticals has strong long-term prospects, it faces short-term hurdles that place it in a medium-risk category.
Rocket Pharmaceuticals Inc. has a mixed financial outlook. While the company is advancing a sustainable pipeline of investigational genetic therapies and has received positive updates such as the Orphan Medicinal Product Designation from the European Commission 7, it also faces significant challenges. The FDA has rejected its gene therapy for a rare immune disorder, asking for more data, which has negatively impacted its stock 8. Additionally, the company has experienced a price target downgrade from multiple financial institutions 9. These factors indicate that while Rocket Pharmaceuticals has strong long-term prospects, it faces short-term hurdles that place it in a medium-risk category.
7: Rocket Pharmaceuticals Receives Orphan Medicinal Product Designation from the European Commission for RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy View Source8: FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock Slides View Source9: Rocket Pharmaceuticals price target lowered to $40 from $49 at CanaccordJuly 3, 2024TipRanksRocket Pharmaceuticals price target lowered to $37 from $39 at BofAJune 30, 2024TipRanksMaintaining Buy on Rocket Pharmaceuticals: Temporary Setback with Strong Long-Term ProspectsJune 28, 2024TipRanksBuy Rating Affirmed for Rocket Pharmaceuticals Amid Manageable FDA HurdleJune 28, 2024TipRanksOptimistic Outlook for Rocket Pharmaceuticals’ KRESLADI Despite FDA Request for Additional CMC InformationJune 28, 2024TipRanks View Source
NO
The provided facts do not indicate that Rocket Pharmaceuticals Inc. is launching any new product offerings. The information focuses on their ongoing programs, community engagements, and financial updates but does not mention any new product launches.
The provided facts do not indicate that Rocket Pharmaceuticals Inc. is launching any new product offerings. The information focuses on their ongoing programs, community engagements, and financial updates but does not mention any new product launches.
Rocket Pharmaceuticals Inc. is strategically focused on advancing its pipeline of investigational genetic therapies from 2024 through 2026, targeting both hematologic diseases and multi-organ disorders. The company's clinical-stage programs include ex vivo lentiviral vector therapies for Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency, as well as in vivo adeno-associated virus therapies for Danon disease and Plakophilin-2 Arrhythmogenic Cardiomyopathy 1011. Despite facing regulatory challenges, such as the FDA's request for additional data on their gene therapy for a rare immune disorder 12, Rocket Pharmaceuticals remains committed to its long-term growth plans and the development of first and best-in-class gene therapy treatment options 13.
10: Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey. View Source11: Rocket Pharmaceuticals, Inc. is a fully integrated, late-stage biotechnology company. The Company is advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Its lentiviral (LV) vector-based gene therapies target hematologic diseases and consist of late-stage programs for Fanconi Anemia, a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte... View Source12: FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock Slides View Source13: Rocket Pharmaceuticals (NASDAQ:RCKT) Is In A Good Position To Deliver On Growth Plans View Source