PKD

 Rocket Pharmaceuticals Announces Pricing of Public Offering of Common Stock

Retrieved on: 
Tuesday, October 4, 2022
Biotechnology, Pharmaceutical, Health, Other Health, 808, NASDAQ, U.S. Securities and Exchange Commission, SEC, Risk, Pyruvate kinase deficiency, Bone marrow failure, PKD, AAV, Morgan Stanley, Private Securities Litigation Reform Act, Fanconi anemia, Danon disease, 2nd Floor (Nina song), Medication, Telephone, LLC, Sumathi Best Television Current Reporting Award, Lists of diseases, J. P. Morgan, Varick Street, FA, Sale, SVB, Genetic disorder, Patient, Heart failure, LVV, State Street, Broadridge Financial Solutions, Attention, Security (finance)

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today the pricing of an underwritten public offering of 6,800,000 shares of its common stock at a public offering price of $14.75 per share.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today the pricing of an underwritten public offering of 6,800,000 shares of its common stock at a public offering price of $14.75 per share.
  • The gross proceeds to Rocket from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $100.3 million.
  • In addition, Rocket has granted the underwriters a 30-day option to purchase up to an additional 1,020,000 shares of its common stock.
  • There can be no assurance that Rocket will be able to complete the public offering on the anticipated terms.

Rocket Pharmaceuticals Announces Proposed Public Offering of Common Stock

Retrieved on: 
Monday, October 3, 2022
Biotechnology, Pharmaceutical, Genetics, Health, 808, NASDAQ, U.S. Securities and Exchange Commission, SEC, Risk, Pyruvate kinase deficiency, Bone marrow failure, PKD, AAV, Morgan Stanley, Private Securities Litigation Reform Act, Fanconi anemia, Danon disease, 2nd Floor (Nina song), Medication, Telephone, LLC, Sumathi Best Television Current Reporting Award, Lists of diseases, J. P. Morgan, Varick Street, FA, Sale, SVB, Genetic disorder, Patient, Heart failure, LVV, State Street, Broadridge Financial Solutions, Attention, Security (finance)

Rocket also intends to grant the underwriters a 30-day option to purchase up to an additional $15 million of shares of its common stock.

Key Points: 
  • Rocket also intends to grant the underwriters a 30-day option to purchase up to an additional $15 million of shares of its common stock.
  • All of the shares in the proposed offering are to be sold by Rocket.
  • The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.
  • There can be no assurance that Rocket will be able to complete the public offering on the anticipated terms.

 Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial for RP-A501 in Danon Disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022

Retrieved on: 
Friday, September 30, 2022
Biotechnology, Pharmaceutical, Health, Clinical Trials, Adult, LV, Safety, Heart Failure Society of America, Pyruvate kinase deficiency, Blood, Cardiac muscle, PKD, Patient, Bone marrow failure, Gene expression, Genetic disorder, NASDAQ, Disease, Death, Risk, Fanconi anemia, COVID-19, Prevalence, AAV, Degenerative disease, Lists of diseases, Heart failure, Glycogen, LVV, Medication, Toxic leukoencephalopathy, SEC, Diastole, Troponin, Freedom, Immunohistochemistry, II, Investor, FDA, Ventricular hypertrophy, Adolescence, Data, BNP, Tissue, Marketing, Blunt cardiac injury, Autophagy, FA, Private Securities Litigation Reform Act, Autophagosome, Quality of life, Male, Heart transplantation, New York Heart Association Functional Classification, KCCQ, Biopsy, Webcast, Pharmaceutical industry, Dentistry, Medical device, Medicine, Vaccine, Danon disease, NYHA

Further, efficacy data from the pediatric patients are following similar or more favorable positive trends as in the adults at a similar timeframe.

Key Points: 
  • Further, efficacy data from the pediatric patients are following similar or more favorable positive trends as in the adults at a similar timeframe.
  • RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure.
  • 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 Chardan 6th Annual Genetic Medicines Conference

Retrieved on: 
Tuesday, September 27, 2022
Biotechnology, Pharmaceutical, Health, Other Health, Risk, Fanconi anemia, Investor, Safety, COVID-19, FA, AAV, Pyruvate kinase deficiency, Danon disease, PKD, Lists of diseases, Private Securities Litigation Reform Act, Westin Hotels & Resorts, Heart failure, Patient, Westin Hotel Cleveland, Bone marrow failure, LVV, Genetic disorder, Medication, NASDAQ, Conference, Marketing, SEC, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the Chardan 6th Annual Genetic Medicines Conference on Tuesday, Oct. 4, at 2:00 p.m.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the Chardan 6th Annual Genetic Medicines Conference on Tuesday, Oct. 4, at 2:00 p.m.
  • 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 .
  • 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.

Autosomal Dominant Polycystic Kidney Disease Market to Surge at a Significant CAGR of 14.5% by 2032 | DelveInsight

Retrieved on: 
Monday, September 19, 2022
Adult, Pediatrics, PKD1, Urology, Research, PKD, Patient, Physician, United Kingdom, PKD2, Kidney, Disease, Mario Negri Institute for Pharmacological Research, AL, Diet, Pharmacological Research, Kadmon Corporation, Prevalence, Back pain, Pain, Polycystic kidney disease, Time-invariant system, Risk, Population, Autosome, Tolvaptan, Otsuka Pharmaceutical, Water, Reata Pharmaceuticals, HIV disease progression rates, Regulus Therapeutics, Ryukyu Kingdom, Cyst, LAS, Genetic counseling, Nephrology, LAR, Headache, Therapy, Galapagos NV, Pharmaceutical industry, Lithium, Tesevatinib, ADPKD, Bardoxolone methyl

LAS VEGAS, Sept. 19, 2022 /PRNewswire/ -- DelveInsight's ADPKD Market Insights report includes a comprehensive understanding of current treatment practices, ADPKD emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU5 (the United Kingdom, Italy, Spain, France, and Germany), Japan].

Key Points: 
  • In addition, the approval and entry of new drugs will change the ADPKD market dynamics in the future.
  • As per DelveInsight estimates, the ADPKDmarket size in the 7MM was approximately USD 1,077 million in 2021.
  • Autosomal dominant polycystic kidney disease also called "adult PKD," is the most common inherited kidney disorder characterized by cyst growth in the kidneys, which increases with disease progression and results in renal failure.
  • Implementing these measures early in the disease should slow the progression of kidney (renal) disease and, to some extent, preserve kidney function.

Rocket Pharmaceuticals to Present Updated Data from Phase 1 Danon Disease Trial for RP-A501 at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022

Retrieved on: 
Friday, September 16, 2022
Science, Cardiology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, Risk, Fanconi anemia, Investor, Safety, Heart Failure Society of America, COVID-19, FA, AAV, Pyruvate kinase deficiency, Danon disease, PKD, Company, Lists of diseases, Society, Private Securities Litigation Reform Act, Heart failure, Portal, Patient, Gene, Cell, Bone marrow failure, LVV, Genetic disorder, Medication, Congress, NASDAQ, Marketing, SEC, Webcast, Pharmaceutical industry

Rocket recently announced positive initial safety data from two pediatric patients in its Phase 1 Danon Disease trial at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).

Key Points: 
  • Rocket recently announced positive initial safety data from two pediatric patients in its Phase 1 Danon Disease trial at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).
  • Title: Safety Profile of the First Pediatric Cardiomyopathy Gene Therapy Trial: RP-A501 (AAV9:LAMP2B) for Danon Disease
    Location: Gaylord National Harbor Convention Center, Washington, D.C.
    Company management will discuss the Danon Disease data via webcast on Sept. 30, 2022, at 8:00 a.m.
  • 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 Morgan Stanley 20th Annual Global Healthcare Conference

Retrieved on: 
Tuesday, September 6, 2022
Biotechnology, Pharmaceutical, Genetics, Health, COVID-19, Safety, Bone marrow failure, NASDAQ, Morgan, Pyruvate kinase deficiency, Fanconi anemia, Genetic disorder, Medication, Risk, Heart failure, PKD, SEC, Lists of diseases, Conference, Danon disease, LVV, Investor, Marketing, Private Securities Litigation Reform Act, FA, AAV, Patient, 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 Companys 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.

XORTX Sponsored Study Selected for Presentation at the American Society of Nephrology – Kidney Week 2022

Retrieved on: 
Thursday, September 1, 2022
COVID-19, Kidney, CEO, Uric acid, Kidney disease, U.S. Securities and Exchange Commission, Fibrosis, GLOBE, Coronavirus, Hyperuricemia, International, Allantoin, University of Colorado Denver School of Public Affairs, Gout, Association, Patient, DS, ASN, Letter, Incidence, PKD, SEC, Nephrology, OXO, Annual report, Cyst, American Society of Nephrology, Therapy, Abstract (summary), NASDAQ, Urate oxidase, Society, TSX Venture Exchange, Quality of life, News, Analysis, Health, Autosome, Chronic pain, University, Polycystic kidney disease, Diabetic nephropathy, Human, Diagnosis, No, Xanthine oxidase, Conference, Form, Peer review, Pharmaceutical industry, Gum arabic, ADPKD, Nasdaq

CALGARY, Alberta, Sept. 01, 2022 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the Company) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease is pleased to announce acceptance of a study abstract for presentation at the American Society of Nephrology (ASN) Kidney Week 2022 meeting to be held the week of November 4, 2022.

Key Points: 
  • CALGARY, Alberta, Sept. 01, 2022 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. ("XORTX" or the Company) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease is pleased to announce acceptance of a study abstract for presentation at the American Society of Nephrology (ASN) Kidney Week 2022 meeting to be held the week of November 4, 2022.
  • Dr. Allen Davidoff, CEO of XORTX, stated, We are pleased to participating in the ASN annual meeting during Kidney Week 2022 with this poster presentation.
  • ASN represents more than 21,000 kidney health professionals working to help people with kidney diseases and their families.
  • Source: https://www.asn-online.org/
    The Kidney Week Conference, being held in Orlando, Florida, is attended by approximately 10,000 kidney professionals from across the globe.

Rocket Pharmaceuticals Reports Second Quarter 2022 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, August 8, 2022
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, Other Health, Advaxis, Survival, Good, SEC, Investment, Schering-Plough, Leadership, Dysplasia, Research, Marketing, Patient, Biotechnology, G&A, PKD, Hemolysis, Medication, Overalls, Lists of diseases, Industry, Private Securities Litigation Reform Act, LVV, Merck, Safety, Kaplan–Meier estimator, MMC, BLA, Science, Celgene, Conference, CMC, Heart failure, Bone marrow failure, FA, CDMO, CD18, COVID-19, Infection, Morgan Stanley, Hematopoietic stem cell transplantation, Novartis, Anemia, Chemistry, Pyruvate kinase deficiency, AAV, FDA, Neutrophil, Genetic disorder, CGMP, Achievement, MedImmune, Good manufacturing practice, Quality of life, Company, Liver injury, Risk, NASDAQ, Fanconi anemia, Adult, Pharmaceutical industry, Fine chemical, Health insurance, Tutoring, Q2, Danon disease

Importantly, we continue to maintain a healthy operational cash runway into the first half of 2024, concluded Dr. Shah.

Key Points: 
  • Importantly, we continue to maintain a healthy operational cash runway into the first half of 2024, concluded Dr. Shah.
  • The originally planned Q3 topline readout for FA was achieved earlier than anticipated in Q2 when the trial met its primary endpoint.
  • Pending health authority interactions, Phase 2 trial planning activities are expected to begin in Q4 of 2022.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

Rocket Pharmaceuticals Appoints Mayo Pujols as Chief Technical Officer and Strengthens Manufacturing Capabilities

Retrieved on: 
Wednesday, July 27, 2022
FDA, Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, Advaxis, Novartis, Chemistry, Medication, Schering-Plough, Industry, CGMP, Fu Foundation School of Engineering and Applied Science, FA, SEC, Pyruvate kinase deficiency, Nationwide Children's Hospital, Risk, Bone marrow failure, Private Securities Litigation Reform Act, Lists of diseases, Genetic disorder, Marketing, Safety, COVID-19, LVV, Applied science, NASDAQ, PKD, Chemical engineering, CTO, Hospital, CDMO, Technology, Leadership, Good manufacturing practice, Stevens Institute of Technology, Merck, MedImmune, Lifting, Patient, Head, Heart failure, CMC, Good, Pharming, Fanconi anemia, AAV, Fine chemical, Pharmaceutical industry, Danon disease, Celgene, Engineering

Mr. Pujols brings nearly three decades of experience, from leadership roles across technical operations, quality operations, validation, process development and Good Manufacturing Practice (cGMP) manufacturing.

Key Points: 
  • Mr. Pujols brings nearly three decades of experience, from leadership roles across technical operations, quality operations, validation, process development and Good Manufacturing Practice (cGMP) manufacturing.
  • He most recently served as Chief Executive Officer of Andelyn Biosciences, a leading gene therapy contract development and manufacturing organization (CDMO) and prior to Andelyn was the Head of Global Cell and Gene Technical Development and Manufacturing for Novartis Pharmaceuticals.
  • As Rockets first CTO, Mr. Pujols will lead the technical operations function and chemistry, manufacturing and controls (CMC) for all lentiviral programs.
  • Prior to joining Rocket, Mr. Pujols was Chief Executive Officer of Andelyn Biosciences, a CDMO affiliated with Nationwide Children's Hospital, where he led its expansion to a full-service gene therapy CDMO.