PKD

Rocket Pharmaceuticals Appoints Jessie Yeung as Vice President of Investor Relations and Corporate Finance

Retrieved on: 
Tuesday, March 8, 2022
Health, Genetics, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, Lists of diseases, Private Securities Litigation Reform Act, IPO, Bone marrow failure, Lifting, Leadership, Patient, MBA, Research, Risk management, Pyruvate kinase deficiency, Heart failure, Genetic disorder, Safety, Fanconi anemia, PKD, Danon disease, LVV, J. P. Morgan, SEC, Company, Columbia Business School, COVID-19, AAV, Wells Fargo, BofA Securities, Carnegie Mellon University, Marketing, Medication, Bank, Business administration, Risk, Corporate finance, FA, NASDAQ, Pharmaceutical industry, Rocket

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Jessie Yeung as Vice President of Investor Relations and Corporate Finance.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Jessie Yeung as Vice President of Investor Relations and Corporate Finance.
  • Ms. Yeung brings more than 15 years of investor relations, corporate finance and capital market experience across industries including the biopharmaceutical and financial sectors.
  • She will be responsible for leading the investor relations function and capital markets strategy.
  • Prior to joining Rocket, Ms. Yeung was the Head of Corporate Finance and Investor Relations at Legend Biotech.

Caraway Therapeutics Expands Scientific Advisory Board with the Appointment of David Clapham, MD, PhD

Retrieved on: 
Wednesday, March 2, 2022
Biotechnology, Pharmaceutical, Health, Other Health, Lists of diseases, Janelia Research Campus, National Academy of Medicine, Harvard Medical School, Ion, Patient, Aldo, Max Planck Society, Research, Max Planck Institute for Experimental Medicine, Bardet–Biedl syndrome, Doctor of Philosophy, Emory University School of Medicine, Hospital, PKD, Coronavirus Scientific Advisory Board (Turkey), Investment, Therapy, Neuroscience, Neurodegeneration, Nobel Prize, Company, BBS, Biophysical chemistry, Polycystic kidney disease, Nephronophthisis, Internal medicine, SAB, Howard Hughes Medical Institute, HHMI, National academy, National, Pharmaceutical industry, Brigham, Caraway, MD

Caraway Therapeutics today announced the appointment of David Clapham, MD, PhD, to the companys Scientific Advisory Board (SAB).

Key Points: 
  • Caraway Therapeutics today announced the appointment of David Clapham, MD, PhD, to the companys Scientific Advisory Board (SAB).
  • I am delighted to welcome Dr. Clapham to Caraway as a scientific advisor, said Martin D. Williams, Chief Executive Officer at Caraway Therapeutics.
  • The combined cutting-edge expertise and wisdom David provides will be a key asset for Caraway as we continue to advance our programs.
  • Dr. Clapham joins Caraways experienced, collaborative, and dedicated Scientific Advisory Board team comprised of tenured biologists and biotech leaders.

National Kidney Foundation Hosts Virtual 9th Annual Kidney Patient Summit

Retrieved on: 
Wednesday, March 2, 2022
People, Dialysis, Risk, Horizon Therapeutics, Chronic kidney disease, Patient, Research, National Kidney Foundation, White, Black, NKF, Kidney disease, Degenerative disease, PKD, Awareness, Congress, African Americans, Alaska Natives, Legislation, Mallinckrodt, COVID-19, Risk factor, Disease, Kidney failure, CEO, Kidney, Obesity, Transplant, Diabetes, Partnership, PKD Foundation, Pharmaceutical industry

WASHINGTON, March 2, 2022 /PRNewswire/ -- Today the National Kidney Foundation (NKF) hosts its 9th Annual Kidney Patient Summit bringing together more than 220 Voices for Kidney Health advocates in partnership with kidney organizations across the United States to virtually meet with their Congressional delegations and share their personal kidney journeys to help inspire action on behalf of the 37 million Americans who have kidney disease, also known as chronic kidney disease.

Key Points: 
  • WASHINGTON, March 2, 2022 /PRNewswire/ -- Today the National Kidney Foundation (NKF) hosts its 9th Annual Kidney Patient Summit bringing together more than 220 Voices for Kidney Health advocates in partnership with kidney organizations across the United States to virtually meet with their Congressional delegations and share their personal kidney journeys to help inspire action on behalf of the 37 million Americans who have kidney disease, also known as chronic kidney disease.
  • Our Voices' advocates will be joined by advocates from Home Dialyzors United , Nephcure Kidney International , the Alport Syndrome Foundation , IGA Nephropathy Foundation of America , and PKD Foundation to advance public policies that will promote better kidney healthfor all Americans.
  • "The Kidney Patient Summit gives our patients and advocates an opportunity to fight passionately for legislation that improves the lives of those with kidney disease," said Kevin Longino, CEO of the National Kidney Foundation and a kidney transplant patient.
  • The National Kidney Foundation (NKF) is the largest, most comprehensive, and longstanding patient-centric organization dedicated to the awareness, prevention, and treatment of kidney disease in the U.S. For more information about NKF, visit www.kidney.org .

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2021 Financial and Operational Results

Retrieved on: 
Thursday, February 24, 2022
Health, Pharmaceutical, Fanconi anemia, Compliance, Empire, Phase II, Awareness, Indoor Living, COVID-19, Company, General counsel, Private Securities Litigation Reform Act, Research, CGMP, Adolescence, Risk, SEC, Investment, Genetic disorder, Pyruvate kinase deficiency, Safety, Lists of diseases, Food, MMC, Quality of life, Bone marrow failure, Orphan drug, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Degenerative disease, NASDAQ, AAV, RTW, G&A, Advanced Therapy Medicinal Product, LP, Heart failure, Good manufacturing practice, Danon disease, EMA, Patient, PKD, History, Medication, FA, Industry, ASH, LVV, Adult, CD18, FDA, Conference, Lifting, Marketing, Anemia, Society, Pharmaceutical industry, Video game, Online gambling, Airline, Internet service provider, EU, Rocket

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial and operational results for the fourth quarter and year ended December 31, 2021.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial and operational results for the fourth quarter and year ended December 31, 2021.
  • Results demonstrated sustained benefit across clinical, functional and biomarker endpoints in all four patients with long-term follow up.
  • Preliminary Phase 1 data and Phase 2 trial initiation activities are expected in the fourth quarter of 2022.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals Announces Participation in 11th Annual SVB Leerink Global Healthcare Conference

Retrieved on: 
Thursday, February 10, 2022
General Health, Health, Genetics, Pharmaceutical, Clinical Trials, NASDAQ, Private Securities Litigation Reform Act, Heart failure, Genetic disorder, LVV, Conference, Danon disease, Lists of diseases, Investor, PKD, Pyruvate kinase deficiency, Marketing, Medication, Patient, Safety, SEC, Lifting, Bone marrow failure, FA, AAV, Fanconi anemia, Risk, COVID-19, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the virtual 11th Annual SVB Leerink Global Healthcare Conference on Wednesday, Feb. 16 at 10 a.m.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the virtual 11th Annual SVB Leerink Global Healthcare Conference on Wednesday, Feb. 16 at 10 a.m.
  • The webcast replay will be available on the Rocket website following the conference.
  • 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.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Spherix Global Insights Embarks on Its Fifth Year of Extensive ADPKD Coverage as the Market Heats Up

Retrieved on: 
Thursday, February 3, 2022
Patient, Overalls, HIV disease progression rates, Physician, Liver, HCP, Nephrology, Ōtsuka, Mark (given name), Neurology, Gastroenterology, Polycystic kidney disease, Hepatotoxicity, Ophthalmology, ADPKD, FDA, NASA RealWorld-InWorld Engineering Design Challenge, Dermatology, PKD, Pharmaceutical industry, Medical device, Galápagos Islands, Tolvaptan

Notably, the percentage of physicians who are "comfortable with the risk-benefit ratio of Jynarque" has dropped substantially since the previous year's ADPKD patient audit.

Key Points: 
  • Notably, the percentage of physicians who are "comfortable with the risk-benefit ratio of Jynarque" has dropped substantially since the previous year's ADPKD patient audit.
  • Spherix's extensive coverage of the polycystic kidney disease (PKD) market since 2018 leverages these annual patient chart audits along with quarterly market tracking studies.
  • Unsurprisingly, nephrologists express desire for a new ADPKD product that has better efficacy, both overall and specifically against cyst growth.
  • Spherix Global Insights is a hyper-focused market intelligence firm that leverages our own independent data and expertise to provide strategic guidance, so biopharma stakeholders make decisions with confidence.

Rocket Pharmaceuticals Highlights Anticipated 2022 Milestones Across Lentiviral and AAV Gene Therapy Clinical Programs

Retrieved on: 
Tuesday, January 11, 2022
Biotechnology, Other Health, Health, Pharmaceutical, Clinical Trials, FA, Patient, PKD, Conference, CD18, Genetic disorder, Danon disease, Private Securities Litigation Reform Act, SEC, COVID-19, MBA, Safety, Risk, NASDAQ, LVV, Pyruvate kinase deficiency, Lists of diseases, Medication, Heart failure, Leukocyte adhesion deficiency, Fanconi anemia, Lifting, Marketing, AAV, Pharming, Bone marrow failure, Pharmaceutical industry, EU

The Rocket team together with our partners made significant progress across our pipeline of first-and best-in-class lentiviral and AAV gene therapies in 2021, said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer of Rocket Pharma.

Key Points: 
  • The Rocket team together with our partners made significant progress across our pipeline of first-and best-in-class lentiviral and AAV gene therapies in 2021, said Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer of Rocket Pharma.
  • 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.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals to Present at the 40th Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Tuesday, January 4, 2022
Health, Genetics, Other Health, Clinical Trials, Research, Science, Safety, Genetic disorder, COVID-19, Investor, AAV, Private Securities Litigation Reform Act, Heart failure, IMO, Lists of diseases, LVV, FA, Medication, Lifting, NASDAQ, Conference, SEC, Danon disease, Fanconi anemia, Pyruvate kinase deficiency, Bone marrow failure, Osteopetrosis, Marketing, Patient, Risk, PKD, Pharmaceutical industry

A live audio webcast of the presentation will be available under Events in the Investors section of the Companys website at https://ir.rocketpharma.com/ .

Key Points: 
  • A live audio webcast of the presentation will be available under Events in the Investors section of the Companys website at https://ir.rocketpharma.com/ .
  • The webcast replay will be available on the Rocket website following the conference.
  • 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.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals Presents Positive Clinical Data from Company’s Lentiviral Gene Therapies for Treatment of Fanconi Anemia, LAD-I and PKD at the 63rd American Society of Hematology (ASH) Annual Meeting

Retrieved on: 
Monday, December 13, 2021
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, CIEMAT, Lists of diseases, HSCT, NASDAQ, Survival, Pyruvate kinase deficiency, Anemia, Risk, MMC, California Institute for Regenerative Medicine, Squamous cell carcinoma, Patient, CIRM, Adult, Blood, FDA, Pneumonia, DNA repair, Diagnosis, DEB, PKD, Splenectomy, FANCA, AAV, Head, Mutation, Regenerative medicine, Pyruvate kinase, Fanconi anemia, Safety, Quality of life, Grant, Birth, ASH, Union, ITGB2, Osteopetrosis, Heart failure, Investor, COVID-19, IMO, SEC, Research, Marketing, DNA, Prevalence, Sepsis, Centro, Danon disease, FA, Șaeș, Death, Life, Private Securities Litigation Reform Act, Genetic disorder, CD18, Mosaic (genetics), Hematopoietic stem cell transplantation, Bone marrow failure, Mortality, LVV, PKLR, Infection, Pharmaceutical industry, Vaccine, Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency, Rocket Pharmaceuticals, Inc., FANCONI ANEMIA, LEUKOCYTE ADHESION DEFICIENCY-I, PYRUVATE KINASE DEFICIENCY, ROCKET PHARMACEUTICALS, INC.

RP-L301 continues to be well tolerated, with no drug product related SAEs or infusion-related complications observed through 12-months post-treatment.

Key Points: 
  • RP-L301 continues to be well tolerated, with no drug product related SAEs or infusion-related complications observed through 12-months post-treatment.
  • At approximately 12-months post-treatment the patient had normalized hemoglobin levels of 14.8 g/dL, compared to a pre-treatment baseline of ~7.0 g/dL.
  • Rocket will host an in-person Investor and Analyst Event that will simultaneously be webcast tomorrow, Dec. 14, at 7:30 a.m.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.

Rocket Pharmaceuticals Appoints Martin L. Wilson as General Counsel and Chief Compliance Officer

Retrieved on: 
Wednesday, December 8, 2021
Science, Research, Pharmaceutical, Health, FDA, Infectious Diseases, Genetics, Clinical Trials, Bone marrow failure, Endo, Lists of diseases, General counsel, Fanconi anemia, Business development, Heart failure, Endo International, SEC, Compliance, Biotechnology, Par, Human resources, Risk, Villanova University, Leadership, Genetic disorder, FA, Safety, Drug development, IMO, Private Securities Litigation Reform Act, PKD, Food, Patient, Danon disease, Corporation, LVV, Human, Pyruvate kinase deficiency, Lifting, Senior, COVID-19, Osteopetrosis, Medication, FDA, Marketing, NASDAQ, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Law, AAV, Juris Doctor, License, Acquisition, Schering-Plough, Assistant, Management, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Martin L. Wilson as General Counsel, Chief Compliance Officer and Senior Vice President.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Martin L. Wilson as General Counsel, Chief Compliance Officer and Senior Vice President.
  • Prior to joining Rocket, Mr. Wilson was General Counsel and Chief Corporate Officer at Ichnos Sciences, where he oversaw Legal, Compliance, Business Development, Human Resources and IT.
  • Before Ichnos, Mr. Wilson served as General Counsel, Chief Compliance Officer, Corporate Secretary and Head of Human Resources at Teligent, Inc.
  • Prior to Teligent, he was Vice President and Assistant General Counsel at Endo Pharmaceuticals following the acquisition of Par Pharmaceuticals.