PKD

Rocket Pharmaceuticals to Present at the 2021 Cell & Gene Meeting on the Mesa

Retrieved on: 
Thursday, October 7, 2021
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, NASDAQ, Lists of diseases, Medication, LVV, Pyruvate kinase deficiency, Bone marrow failure, PST, FA, Mesa, IMO, PKD, Danon disease, Osteopetrosis, Fanconi anemia, Cell, AAV, CA, Patient, Heart failure, Genetic disorder, Vaccine

The Rocket Pharmaceuticals presentation will take place on Tuesday, October 12 at 2:15 p.m. PST.

Key Points: 
  • The Rocket Pharmaceuticals presentation will take place on Tuesday, October 12 at 2:15 p.m. PST.
  • 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.
  • The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.

Rocket Pharmaceuticals to Participate in the Chardan Virtual 5th Annual Genetic Medicines Conference

Retrieved on: 
Thursday, September 30, 2021
Other Health, Health, Genetics, Pharmaceutical, Clinical Trials, Heart failure, PKD, FA, Target, Conference, NASDAQ, Webcast, Patient, Bone marrow failure, Medication, Danon disease, Lists of diseases, Pyruvate kinase deficiency, LVV, Genetic disorder, Heart, Osteopetrosis, AAV, IMO, Fanconi anemia, 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 announced that Gaurav Shah, M.D., chief executive officer, will participate in a fireside chat on Tuesday, October 5, 2021, at 10:30 am ET at the Chardan Virtual 5th Annual Genetic Medicines Conference.

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 announced that Gaurav Shah, M.D., chief executive officer, will participate in a fireside chat on Tuesday, October 5, 2021, at 10:30 am ET at the Chardan Virtual 5th Annual Genetic Medicines Conference.
  • The live webcast of the fireside chat will be accessible via Rockets website on the Events page.
  • An archived copy of the webcast will be available on the Rocket website for 30 days after the event.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders.

Rocket Pharmaceuticals Presents Clinical Data from RP-A501 Trial in Danon Disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021

Retrieved on: 
Tuesday, September 14, 2021
Science, Cardiology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, Autophagosome, Medication, IMO, Tissue, Genetic disorder, Cardiac muscle, Patient, Immunohistochemistry, Vacuole, Marketing, LVV, Private Securities Litigation Reform Act, Autophagy, Lists of diseases, Glycogen, Danon disease, Fanconi anemia, Safety, BNP, NASDAQ, Osteopetrosis, COVID-19, Heart Failure Society of America, Bone marrow failure, Heart failure, Lifting, Male, FA, PKD, Risk, AAV, SEC, Pyruvate kinase deficiency, Prevalence, Pharmaceutical industry, Vaccine, RP-A501, Danon Disease, Rocket Pharmaceuticals, Inc., RP-A501, DANON DISEASE, ROCKET PHARMACEUTICALS, INC.

Steroid-induced myopathy was observed in two of the three patients >2 weeks after dosing, which also resolved.

Key Points: 
  • Steroid-induced myopathy was observed in two of the three patients >2 weeks after dosing, which also resolved.
  • In these two patients, a substantial improvement of a key marker of heart failure, B-type natriuretic peptide (BNP), was also observed.
  • 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.

Rocket Pharmaceuticals Announces Upcoming Danon Disease Presentation at the Heart Failure Society of America Annual Scientific Meeting 2021

Retrieved on: 
Friday, September 10, 2021
Biotechnology, Pharmaceutical, Health, Clinical Trials

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that previously disclosed data from the ongoing Phase 1 trial of RP-A501 in Danon Disease will be presented in a late-breaking oral presentation at the upcoming Heart Failure Society of America (HFSA) Annual Scientific Meeting 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 announces that previously disclosed data from the ongoing Phase 1 trial of RP-A501 in Danon Disease will be presented in a late-breaking oral presentation at the upcoming Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021.
  • The meeting will take place September 10-13, 2021 at the Gaylord Rockies Hotel and Conference Center in Aurora, Colorado.
  • Details for Rockets oral presentation are as follows:
    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.

Rocket Pharmaceuticals Appoints Isabel Carmona, J.D., as Chief Human Resources Officer

Retrieved on: 
Tuesday, September 7, 2021
Professional Services, Health, Genetics, Human Resources, Pharmaceutical, Biotechnology, Rocket Pharmaceuticals Leadership, Rocket Pharmaceuticals, Inc., ROCKET PHARMACEUTICALS LEADERSHIP, ROCKET PHARMACEUTICALS, INC.

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 Isabel Carmona, J.D., as chief human resources 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 Isabel Carmona, J.D., as chief human resources officer and senior vice president.
  • I am delighted to welcome Isabel to Rocket and believe her rich experience leading organizational growth at life science companies will be a tremendous asset as we continue our exciting growth trajectory, said Gaurav Shah, M.D., chief executive officer of Rocket Pharma.
  • Prior to joining Rocket, Ms. Carmona was chief human resources officer of Ichnos Sciences.
  • Prior to that, she served in positions of increasing leadership in the global operations and human resources teams at Teva Pharmaceuticals and Shire Pharmaceuticals.

Imara to Present at Upcoming Investor Conferences

Retrieved on: 
Friday, September 3, 2021
Patient, PDE9, Citigroup, Sickle cell disease, Lists of diseases, Morgan, Morgan Stanley, Nasdaq, Conference, Company, Thalassemia, ITP, PKD, GLOBE, Therapy, Pharmaceutical industry, Medical device

Dr. Ballal will give a pre-recorded presentation at the H.C. Wainwright 23rd Annual Global Investment Conference which will be made available on September 13, 2021 at 7:00 a.m.

Key Points: 
  • Dr. Ballal will give a pre-recorded presentation at the H.C. Wainwright 23rd Annual Global Investment Conference which will be made available on September 13, 2021 at 7:00 a.m.
  • Dr. Ballal and Dr. Attie will participate in a fireside chat at the Morgan Stanley 19th Annual Global Healthcare Conference on September 15, 2021 at 11:00 a.m.
  • Imara Inc. is a clinical-stage biotechnology company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin and other serious diseases.
  • Imara is currently advancing IMR-687, a highly selective, potent small molecule inhibitor of PDE9 that is an oral, once-a-day, potentially disease-modifying treatment for sickle cell disease and beta-thalassemia.

Rocket Pharmaceuticals Announces $26.4 Million Private Placement

Retrieved on: 
Monday, August 30, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Company, Private Securities Litigation Reform Act, Investment, Securities Act of 1933, Genetic disorder, U.S. Securities and Exchange Commission, LVV, AAV, SEC, Research, Bone marrow failure, IMO, RTW, PKD, Osteopetrosis, Marketing, Fanconi anemia, LP, Medication, NASDAQ, Prospectus, Safety, Pyruvate kinase deficiency, Patient, Sale, FA, Lists of diseases, Annual report, COVID-19, Heart failure, Danon disease, Pharmaceutical industry

The private placement is expected to close on or about August 31, 2021, subject to the satisfaction of customary closing conditions.

Key Points: 
  • The private placement is expected to close on or about August 31, 2021, subject to the satisfaction of customary closing conditions.
  • Rocket expects to use the net proceeds from the private placement to continue to advance and expand its pipeline of product candidates, for research and development expenses and for working capital.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that aim to 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 Announces FDA Lifts Clinical Hold on Danon Disease Trial of RP-A501

Retrieved on: 
Monday, August 16, 2021
Other Health, Research, General Health, Pharmaceutical, Genetics, Clinical Trials, Science, Cardiology, Biotechnology, FDA, Other Science, Health, Tissue, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NASDAQ, Bone marrow failure, Danon disease, Adult, Male, Genetic disorder, FA, Company, Risk, Food, Private Securities Litigation Reform Act, Lists of diseases, Death, COVID-19, Program, LVV, SEC, Pyruvate kinase deficiency, Autophagy, FDA, Safety management system, Gene expression, PKD, Fanconi anemia, Patient, Osteopetrosis, Lifting, Marketing, IMO, Medication, Heart failure, Glycogen, Safety, AAV, Cardiac muscle, Autophagosome, Prevalence, Pharmaceutical industry, Vaccine, RP-A501, Danon Disease, Rocket Pharmaceuticals, Inc., RP-A501, DANON DISEASE, ROCKET PHARMACEUTICALS, INC.

We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial.

Key Points: 
  • We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial.
  • We look forward to progressing this critical work on behalf of all Danon patients, said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.
  • Rockets Danon Disease program was placed on clinical hold by the FDA in May of 2021 to modify the study protocol and other supporting documents with revised guidelines for patient selection and safety management.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.

Regulus Therapeutics Presents Additional Data from its Autosomal Dominant Polycystic Kidney Disease (ADPKD) Program at PKD Connect 2021

Retrieved on: 
Friday, June 25, 2021
Branches of biology, Medical specialties, Nephrology, Ion channels, Kidney diseases, Polycystin 1, Autosomal dominant polycystic kidney disease, Polycystin, PKD, Creatinine, PC1, Blood urea nitrogen

Levels of PC1 and PC2 have previously been shown to be inversely correlated with disease severity.

Key Points: 
  • Levels of PC1 and PC2 have previously been shown to be inversely correlated with disease severity.
  • The poster also describes new data from relevant preclinical models showing treatment with RGLS4326 results in increased gene and polycystin levels in vitro.
  • In addition,improvements in key disease markers including serum creatinine and BUN were demonstrated in the Pkd1(F/RC) mouse model that harbors Pkd1 mutation equivalent to human ADPKD.
  • These and other risks are described in additional detail in Regulus' filings with the Securities and Exchange Commission.

Regulus Therapeutics Announces Incremental Update of Autosomal Dominant Polycystic Kidney Disease (ADPKD) Program

Retrieved on: 
Friday, May 21, 2021
Ion channels, Polycystin, PKD, Food and Drug Administration, Forward-looking statement, Branches of biology, Articles, Biology

Regulus believes these data demonstrate that RGLS4326 engages the target miR-17 leading to increased expression of the PKD1 and PKD2 genes and the resultant increases in polycystins\' levels.

Key Points: 
  • Regulus believes these data demonstrate that RGLS4326 engages the target miR-17 leading to increased expression of the PKD1 and PKD2 genes and the resultant increases in polycystins\' levels.
  • Regulus plans to discuss its approach to addressing the remaining partial clinical hold requirements with FDA in mid-2021.
  • These forward-looking statements are based upon Regulus\' current expectations and involve assumptions that may never materialize or may prove to be incorrect.
  • All forward-looking statements contained in this press release speak only as of the date on which they were made.