Autophagosome

 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.

Gain Therapeutics, Inc. Reports First Quarter 2022 Financial Results and Business Update

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Monday, May 16, 2022
Clinical trial, Autophagosome, Research, Survival, Nasdaq, GD, Gaucher's disease, Neurodegeneration, Patient, GBA1, Alpha-synuclein, Information technology, Trial of the century, Therapy, Macrophage, XXVII, AD, Hope, Neuron, Brain, CNS, Lysosome, Conference, GBA, Lead, Parkinson, Program, WORLD, Liver support system, Allosteric regulation, PD, GAAP, Drug discovery, Potency (pharmacology), GLOBE, Medical device, Pharmaceutical industry, Aquaculture, Airline, WT, Gaucher

BETHESDA, Md., May 16, 2022 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company transforming drug discovery with its proprietary computational discovery platform identifying novel allosteric binding sites and creating small molecule treatments, today announced its financial results for the first quarter ended March 31, 2022, and highlighted recent corporate accomplishments.

Key Points: 
  • This has been a productive quarter for Gain, as we continued to demonstrate the broad reaching potential of our SEE-Tx platform, said Eric Richman, Chief Executive Officer of Gain Therapeutics.
  • Our strategy of exploring previously unidentified allosteric binding sites and targeting them for therapeutic benefit has proved promising.
  • In March, Gain presented, Targeting Glucocerebrosidase with Structurally Targeted Allosteric Regulators Corrects Abnormal Phenotypes in Models of Parkinsons Disease.
  • Cash and cash equivalents were $34.34 million as of March 31, 2022 compared to $36.88 million at December 31, 2021.

Gain Therapeutics Presents Preclinical Data on its Structurally Targeted Allosteric Regulators in GBA1 Gaucher Disease at the WORLDSymposium 2022

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Tuesday, February 8, 2022
GLOBE, Glycogen storage disease, Nervous system, GBA1, WT, Gaucher's disease, Protein, Star, Research, GBA, Macrophage, Treatment, GD, IND, Therapy, Lists of diseases, Manchester Grand Hyatt Hotel, Cell death, The Michael J. Fox Foundation, Autophagosome, Lysosomal storage disease, Liver, Spleen, MJFF, Traffic barrier, Nasdaq, Drug discovery, Bone marrow, Neuron, Forward-looking statement, Pharmaceutical industry, Aroma compound

BETHESDA, Md., Feb. 08, 2022 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (Gain),a biotechnology company transforming the drug discovery paradigm with structurally targeted allosteric regulators (STARs) identified with its proprietary computational discovery platform, today presented new pre-clinical data from its Gaucher Disease (GD) program.

Key Points: 
  • BETHESDA, Md., Feb. 08, 2022 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (Gain),a biotechnology company transforming the drug discovery paradigm with structurally targeted allosteric regulators (STARs) identified with its proprietary computational discovery platform, today presented new pre-clinical data from its Gaucher Disease (GD) program.
  • The presentation titled Development of Structurally Targeted Allosteric Regulators for the Treatment of Neuronopathic Gaucher Disease demonstrated the following results:
    Orally bioavailable and brain-penetrant lead STARs have shown promising effects in relevant in vitro models of GD.
  • Gaucher Disease is a lysosomal storage disease characterized by deficient activity of the glucocerebrosidase enzyme (GCase) encoded by the GBA1 gene.
  • Gain Therapeutics, Inc. is transforming the drug discovery paradigm with structurally targeted allosteric regulators identified with its proprietary computational discovery platform SEE-Tx.

Rocket Pharmaceuticals Announces Danon Disease Presentation at the American Heart Association (AHA) Scientific Sessions 2021

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Monday, November 8, 2021
General Health, Health, Pharmaceutical, Clinical Trials, Cardiology, Lifting, Safety, Autophagosome, Glycogen, AHA, Private Securities Litigation Reform Act, Tissue, IMO, Lists of diseases, AAV, Pyruvate kinase deficiency, NASDAQ, American Heart Association, LVV, COVID-19, Danon disease, Medication, Cardiac muscle, Osteopetrosis, Risk, Heart failure, Fanconi anemia, Autophagy, SEC, PKD, Male, Genetic disorder, Bone marrow failure, FA, Patient, Prevalence, Marketing, Pharmaceutical industry, Vaccine, RP-A501, Danon Disease, Rocket Pharmaceuticals, Inc., RP-A501, DANON DISEASE, 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 that updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease will be presented at the American Heart Association (AHA) Scientific Sessions 2021 taking place virtually November 13-15, 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 updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease will be presented at the American Heart Association (AHA) Scientific Sessions 2021 taking place virtually November 13-15, 2021.
  • RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure.
  • Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.

Deciphera Presents Preclinical Data from DCC-3116 Program at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics

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Thursday, October 7, 2021
Biotechnology, Pharmaceutical, Health, NASDAQ, Private Securities Litigation Reform Act, Large-cell lung carcinoma, Pharmacokinetics, EGFR, Autophagosome, Drug resistance, GIST, Conference, BRAF, LLC, COVID-19, Survival, LinkedIn, Growth, Safety, Cancer, Feedback, United States, RAS, KRAS, SEC, Melanoma, ULK1, U.S. Securities and Exchange Commission, Gefitinib, ULK, MEK, AACR, Autophagy, United, Â, RAF, Afatinib, Patient, Neoplasm, ULK2, View, Colorectal cancer, NRAS, Phosphorylation, Pharmaceutical industry, DCC-3116, Deciphera Pharmaceuticals, DCC-3116, DECIPHERA PHARMACEUTICALS

These results reinforce the broad potential of autophagy inhibition as a mechanism to address the challenge of drug resistance in the treatment of cancer.

Key Points: 
  • These results reinforce the broad potential of autophagy inhibition as a mechanism to address the challenge of drug resistance in the treatment of cancer.
  • The poster presentation is available on-demand via the meeting website and on the Companys website at www.deciphera.com/presentations-publications .
  • Deciphera is a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer.
  • Deciphera, the Deciphera logo, QINLOCK, and the QINLOCK logo are registered trademarks of Deciphera Pharmaceuticals, LLC.

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
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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 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.