Cardiac muscle

Shionogi & Co., Ltd. and Maze Therapeutics, Inc. Announce Exclusive Worldwide License Agreement for MZE001, a Novel Therapeutic Candidate for the Treatment of Pompe Disease

Retrieved on: 
Freitag, Mai 10, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Other Health, Patient, Glycogen, Agonal respiration, Intellectual property, Hart–Scott–Rodino Antitrust Improvements Act, Food, Cardiac muscle, Business plan, FDA, Therapy, HSR, Disease, Act, Orphan drug, Tissue, Head, GAA, Plasma protein binding, Pharmaceutical industry

Under the terms of the agreement, Shionogi has acquired exclusive worldwide rights for MZE001 as well as related programs and intellectual property.

Key Points: 
  • Under the terms of the agreement, Shionogi has acquired exclusive worldwide rights for MZE001 as well as related programs and intellectual property.
  • The required 30-day waiting period outlined in United States Hart-Scott-Rodino (HSR) Act has expired, and the transaction is completed.
  • “This agreement is a strong strategic fit for Shionogi.
  • The FDA grants Orphan Drug designation to prevent, diagnose or treat a rare disease or condition.

Affinia Therapeutics Presents Preclinical Data for Genetic Cardiomyopathy and Sporadic ALS Programs with Novel Cardiotropic and BBB-Penetrant AAV Capsids at the American Society of Gene & Cell Therapy 2024 Annual Meeting

Retrieved on: 
Freitag, Mai 10, 2024
Traffic barrier, Patient, Cell, Abstract (summary), Transcytosis, Toxicity, Liver, Cardiac muscle, Capsid, AAV, NHP, BAG3, Therapy, Safety, ALS, Heart, Ballroom, MBA, Senior, MD, Ataxin-2, Spinal cord, Research, Gene, HEK293, Neuron, CNS, BBB, DRG, Brain cell, Computational science, Brain, Society, Science, Cardiomyopathy, Tropism, Skeletal muscle, Central nervous system, Annual general meeting, Vaccine

WALTHAM, Mass., May 10, 2024 /PRNewswire/ -- Affinia Therapeutics ("Affinia"), an innovative gene therapy company with a proprietary platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies for rare and prevalent devastating diseases, today announced the presentation of new preclinical data on its novel AAV capsids for genetic cardiomyopathies and diseases of the central nervous system (CNS) such as amyotrophic lateral sclerosis (ALS), as well as the Company's high-yield manufacturing process. This research will be presented in oral sessions today at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting, being held May 7-11, 2024 in Baltimore, MD and virtually.

Key Points: 
  • This research will be presented in oral sessions today at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting, being held May 7-11, 2024 in Baltimore, MD and virtually.
  • Preclinical efficacy and safety results with a cardiotropic vector encoding for the BAG3 protein provide proof-of-concept data to support the expansion to multiple cardiomyopathies.
  • "Compared with AAV9, our next-generation bespoke capsids demonstrate superior cardiac transduction while detargeting the liver and DRG in single-clone NHP studies.
  • Affinia has also been invited to present on the rational design of cardiotropic capsids that detarget the liver and DRG.

Affinia Therapeutics to Present Data on Novel Cardiotropic and BBB-Penetrant AAV Capsids and Preclinical Efficacy and Safety in Genetic Cardiomyopathies and Sporadic ALS at the American Society of Gene & Cell Therapy 2024 Annual Meeting

Retrieved on: 
Montag, April 22, 2024
DRG, Heart, Traffic barrier, Computational science, Safety, Abstract (summary), Tropism, Cardiac muscle, MD, Structure, Diagram, Cell, BAG3, AAV, Skeletal muscle, CNS, Senior, Synthetic media, Liver, Gene, Society, ALS, Ataxin-2, BBB, Toxicity, Central nervous system, Messenger, Generative, Transcytosis, Annual general meeting, Therapy, Vaccine

Affinia's novel capsids have favorable manufacturing yields and levels of preexisting population immunity.

Key Points: 
  • Affinia's novel capsids have favorable manufacturing yields and levels of preexisting population immunity.
  • "We are excited to unveil data on our next-generation bespoke capsids that demonstrate robust cardiac transduction while detargeting the liver and DRG.
  • One oral presentation showcases data from Affinia's novel, BBB-penetrant AAV capsid with a potential therapeutic application to sporadic ALS.
  • The first poster showcases data that confirm the novel cardiotropic AAV capsid's safety and therapeutic efficacy in an animal model of cardiac dysfunction with a potential therapeutic application in genetic cardiomyopathies.

ZOLL Receives Innovative Technology Contract from Vizient for TherOx SuperSaturated Oxygen (SSO2) Therapy

Retrieved on: 
Dienstag, April 2, 2024
Surgery, Medical Devices, Hospitals, Health, Cardiology, Angioplasty, Asahi Kasei, Patient, Cardiac muscle, Facial muscles, Mortality, Splenic infarction, AUC, STEMI, LAD, Heart, Safety, Patient safety, FDA, Nursing

ZOLL®, an Asahi Kasei company that manufactures medical devices and related software solutions, announced today that it has received an Innovative Technology contract from Vizient, Inc. , the nation’s largest provider-driven healthcare performance improvement company.

Key Points: 
  • ZOLL®, an Asahi Kasei company that manufactures medical devices and related software solutions, announced today that it has received an Innovative Technology contract from Vizient, Inc. , the nation’s largest provider-driven healthcare performance improvement company.
  • Innovative Technology contracts are recommended after review and interaction with products submitted through Vizient’s Innovative Technology Program.
  • Through its Innovative Technology Program , Vizient works with customer-led councils and task forces to evaluate products for their potential to bring real innovation to healthcare.
  • Vizient may award a contract to products deemed worthy of the Innovative Technology designation outside of the competitive bid cycle.

Capricor Therapeutics Presents at 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

Retrieved on: 
Mittwoch, März 6, 2024
MRI, Therapy, SAN, Biotechnology, Skeleton, FDA, DMD, Duchenne muscular dystrophy, Conference, Patient, OLE, Cardiac muscle, Exosome, MDA, LVEF, Episcopal conference, PUL, Pharmaceutical industry

SAN DIEGO, March 06, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company will present the positive 24-month results from its HOPE-2 open-label extension (OLE) study with lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD) at this year’s MDA Clinical and Scientific Conference which is taking place in Orlando, Florida from March 3-6, 2024.

Key Points: 
  • SAN DIEGO, March 06, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company will present the positive 24-month results from its HOPE-2 open-label extension (OLE) study with lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD) at this year’s MDA Clinical and Scientific Conference which is taking place in Orlando, Florida from March 3-6, 2024.
  • CAP-1002 revealed clinically meaningful improvements in ameliorating cardiac function.
  • “We are pleased to be presenting our positive long-term open label extension data at this year’s MDA Conference,” said Linda Marbán, Ph.D., chief executive officer of Capricor.
  • These two-year results suggest that CAP-1002 has the potential to slow the decline of DMD progression and speaks to the potential long-term benefit for patients.

Groundbreaking Study Finds Whole Blood and Plasma Nutrient Tests Should Not be Used for Diagnosing Cardiac Issues in Dogs

Retrieved on: 
Donnerstag, März 7, 2024
Multimedia, Animal, Dog, PAS, Veterinarian, Cardiology, ACAS, University, Associate, Patient, Journal, Cardiac muscle, Heart, Plasma, Research, Taurine

BENTONVILLE, Ark., March 7, 2024 /PRNewswire/ -- Animal nutritionists and veterinarians from BSM Partners, the largest pet care research and consulting firm, and the Chief of Cardiology at the University of Missouri's Veterinary Health Center, published the results of a seven-month study that found no correlation between plasma, whole blood, skeletal and cardiac muscle taurine concentrations in dogs. This development indicates diagnoses of cardiac disease in dogs must not rely solely on blood tests. The research appeared in a peer-reviewed article in the Journal of Animal Physiology and Animal Nutrition.

Key Points: 
  • This development indicates diagnoses of cardiac disease in dogs must not rely solely on blood tests.
  • The research appeared in a peer-reviewed article in the Journal of Animal Physiology and Animal Nutrition .
  • "This particular research found that blood tests are not a reliable indicator for nutrient levels in the heart," said Dr. Sydney McCauley PhD, PAS, Dpl.
  • Blood samples were collected every 30 days throughout the seven-month study and cardiac endomyocardial and skeletal muscle biopsies were performed at the beginning and end of the study.

Cardiol Therapeutics Announces Study Results Presented at the 2023 Annual Meeting of the European Society of Cardiology Working Group on Myocardial and Pericardial Diseases

Retrieved on: 
Donnerstag, November 16, 2023
Pericarditis, Cardiology, Cannabidiol, University, American Heart Association, Research, CRDL, Working Group, Health, Therapy, Cardiac muscle, MMT, Pericardium, Agent handling, Knowledge, Poster, Fibrosis, TSX, Diagnosis, Facial muscles, Heart failure, Wound healing, Pharmaceutical industry

An abstract summarizing these results was submitted by the Company's international research collaborators from the University of Virginia and Houston Methodist DeBakey Heart & Vascular Center to the 2023 Annual Meeting of the European Society of Cardiology Working Group on Myocardial and Pericardial Diseases ("MPD2023") held on November 15 and 16, 2023 in Belgrade, Serbia.

Key Points: 
  • An abstract summarizing these results was submitted by the Company's international research collaborators from the University of Virginia and Houston Methodist DeBakey Heart & Vascular Center to the 2023 Annual Meeting of the European Society of Cardiology Working Group on Myocardial and Pericardial Diseases ("MPD2023") held on November 15 and 16, 2023 in Belgrade, Serbia.
  • The poster entitled "Cannabidiol Inhibits the Mesothelial to Mesenchymal Transition in Experimental Pericarditis" was presented for general viewing within the poster sessions of the MPD2023 Scientific Programme.
  • The 2023 Annual Meeting of the European Society of Cardiology Working Group on Myocardial and Pericardial Diseases brings together a collaborative group dedicated to advancing knowledge, research, and clinical practises related to myocardial and pericardial diseases.
  • The Working Group aims to develop and disseminate guidelines, recommendations, and advancements in the field, ultimately enhancing patient care and outcomes for individuals affected by myocardial and pericardial diseases within the European and global medical community.

Lexeo Therapeutics Granted FDA Fast Track Designation and Orphan Drug Designation for LX2020, an AAV-Based Gene Therapy Candidate for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

Retrieved on: 
Montag, Dezember 18, 2023
Food, FDA, PKP2, Therapy, ACM, Orphan drug, Cardiac muscle, Arrhythmogenic cardiomyopathy, Arrhythmia, Biomarker, Patient, Safety, Disorder, Fast Track, Pharmaceutical industry

“Receiving both Orphan Drug and Fast Track designations from the FDA for LX2020 further validates the importance of progressing a potential one-time treatment option for patients suffering from PKP2-ACM,” commented R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics.

Key Points: 
  • “Receiving both Orphan Drug and Fast Track designations from the FDA for LX2020 further validates the importance of progressing a potential one-time treatment option for patients suffering from PKP2-ACM,” commented R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics.
  • Preliminary efficacy measures will evaluate myocardial protein expression, biomarkers measuring cardiac structure and function, and arrhythmia burden.
  • Long-term safety and efficacy will be evaluated for an additional four years following completion of the initial trial.
  • Fast Track designation allows more frequent FDA interactions to facilitate development and expedite the review process for novel drug candidates that treat serious or life-threatening diseases and address unmet medical needs.

Ethris and Heqet Therapeutics Announce Collaboration to Develop RNA-based Therapeutics for Heart Attack and Heart Failure

Retrieved on: 
Montag, November 13, 2023
Snap, Therapy, Small RNA, Regeneration, Tissue, Biotechnology, Vaccination, Patient, Myocardial infarction, Regenerative medicine, Vaccine, Heqet, Heart failure, Inhalation, LNP, Cardiac muscle, Death, Cell, RNA, Pharmaceutical industry

Under the terms of the agreement, Heqet Therapeutics will lead the development of the program, while Ethris will provide its proprietary Stabilized NanoParticle (SNaP) LNP platform to deliver ncRNA payloads to the heart tissue.

Key Points: 
  • Under the terms of the agreement, Heqet Therapeutics will lead the development of the program, while Ethris will provide its proprietary Stabilized NanoParticle (SNaP) LNP platform to deliver ncRNA payloads to the heart tissue.
  • "Our RNA delivery platform was designed to deliver any type of RNA payload to a broad range of tissues," said Dr. Carsten Rudolph, CEO of Ethris.
  • Under the terms of the collaboration agreement, Ethris and Heqet will operate under a comprehensive commercial agreement, which encompasses various milestone and royalty payments.
  • By harnessing Ethris' SNaP LNP platform for precise delivery, Heqet aims to test the efficacy of its ncRNA candidates to stimulate cardiac regeneration in patients with myocardial infarction and heart failure.

Rejuvenate Bio Announces Preclinical Data for Gene Therapy Candidate RJB-0402 for the Treatment of Arrhythmogenic Cardiomyopathy

Retrieved on: 
Montag, Oktober 9, 2023
Genetics, Clinical Trials, Cardiology, Biotechnology, Other Health, Health, Pharmaceutical, Other Science, Science, Disease, Cardiac cycle, HIV disease progression rates, Arrhythmia, Rejuvenation, Research, Trial of the century, B cell, DSM-IV codes, MD, Liver, ACM, IND, Heart, Sudden death, Mutation, FDA, Heart failure, AAV, Gene, ARVC, Genetic disorder, PVC, DSP, SADS, Arrhythmogenic cardiomyopathy, Cardiac muscle, FGF21, Patient, Quality of life, Vaccine, Medical device, Pharmaceutical industry

Rejuvenate Bio , today announced new preclinical efficacy data for RJB-0402, a gene therapy targeting key disease-mediating pathways.

Key Points: 
  • Rejuvenate Bio , today announced new preclinical efficacy data for RJB-0402, a gene therapy targeting key disease-mediating pathways.
  • RJB-0402 demonstrated efficacy in a mouse model of arrhythmogenic cardiomyopathy (ACM), an inherited disease caused by mutations in one of several genes encoding desmosomal proteins.
  • “What makes RJB-0402 especially promising is its superior performance in the ACM nonclinical model, outperforming gene replacement therapies in reducing PVCs.
  • The initial clinical study for RJB-0402 will focus on patients with Desmoplakin-related arrhythmogenic cardiomyopathy (DSP ACM) at high risk of life-threatening ventricular arrhythmias and sudden cardiac death.