Liver

Elevate Your Sleep Game This World Health Day with LAC

Retrieved on: 
torsdag, april 25, 2024
Relaxation, Magnesium, Liver disease, Risk, Sleep disorder, Health, Sleep, World Health Day, Insomnia, Melatonin, Liver, Valerian (herb), Pharmaceutical industry

Only 1 in 4 Singaporeans have an ideal sleep cycle of 7 hours or more, with insomnia being a highly common sleep disorder that affects up to 15.3% of the local population.

Key Points: 
  • Only 1 in 4 Singaporeans have an ideal sleep cycle of 7 hours or more, with insomnia being a highly common sleep disorder that affects up to 15.3% of the local population.
  • LAC understands the importance of the role that sleep plays in our day to day overall wellbeing.
  • In conjunction with World Health Day, LAC is looking to raise awareness among Singaporeans from all walks of life who are struggling with having healthy sleep habits, highlighting the importance of having adequate sleep and how it enhances their overall well-being.
  • Engineered with triple-layer technology, it ensures both immediate and sustained release, fostering not only relaxation but also an elevated sleep experience.

Kate Therapeutics Unveils Platform and Pipeline Progress at American Society of Gene & Cell Therapy 2024 Annual Meeting

Retrieved on: 
torsdag, maj 9, 2024
Mortality, FDA, University, Facioscapulohumeral muscular dystrophy, Mouse, RNA interference, Safety, Tissue, Heart, Animal, Death, MD, Primate, Cell, Duchenne, Science, Disease, Rockefeller University, Patient, Liver, Society, Gene, X-linked myotubular myopathy, Pediatrics, Gene expression, Judgement, Duchenne muscular dystrophy, Capsid, Annual general meeting, SAN, Muscular dystrophy, Vaccine

SAN DIEGO, May 9, 2024 /PRNewswire/ -- Kate Therapeutics Inc. ("KateTx"), a next-generation gene therapy company, will present detailed preclinical efficacy and safety results on its pipeline and platform at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting being held May 7-11, 2024, in Baltimore, MD and virtually. The results support the best-in-class potential of KateTx's newest generation of skeletal muscle- and heart-targeted, liver de-targeted engineered MyoAAV capsids and gene regulation technology to treat Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), and other devastating genetic skeletal muscle and heart diseases.

Key Points: 
  • KateTx remains on track to select development candidates for additional muscle and cardiac programs in the near term.
  • "KateTx has made significant advances over the past 12 months," said Kevin Forrest, Ph.D., co-founder, president and CEO of KateTx.
  • KateTx's results show the potential to give a one-time gene therapy that potently suppresses DUX4 whenever it is expressed in skeletal muscles.
  • In vitro, the company's MyoAAV-LD mediated RNAi gene therapy candidate showed potent knockdown of DUX4 in FSHD patient myotubes with no off-target effects.

Tempest Reports First Quarter 2024 Financial Results and Provides Business Update

Retrieved on: 
torsdag, maj 9, 2024
Immunotherapy, FDA, Society, Nivolumab, Research, National Cancer Institute, Bevacizumab, PD-1, ASCO, Journal, Therapy, Liver, Patient, NCI, SITC, RCC, AACR, Kidney cancer, FFA, Growth, HCC, Pharmaceutical industry

BRISBANE, Calif., May 09, 2024 (GLOBE NEWSWIRE) -- Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-classi targeted and immune-mediated therapeutics to fight cancer, today reported financial results for the quarter ended March 31, 2024, and provided a corporate update.

Key Points: 
  • BRISBANE, Calif., May 09, 2024 (GLOBE NEWSWIRE) -- Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-classi targeted and immune-mediated therapeutics to fight cancer, today reported financial results for the quarter ended March 31, 2024, and provided a corporate update.
  • These data further support the clinical benefit observed in the TPST-1120 Phase 1 data presented in an oral presentation at ASCO 2022.
  • Tempest ended the quarter with $32.3 million in cash and cash equivalents, compared to $39.2 million on December 31, 2023.
  • Based on its current cash and operating plan, Tempest expects to have sufficient resources to fund operations into the second quarter of 2025.

Chemomab Therapeutics Announces First Quarter 2024 Financial Results and Provides Corporate Update

Retrieved on: 
torsdag, maj 9, 2024
Mortality, FDA, Research and development, TEL, Systemic scleroderma, Nature, Capital, Publication, CMMB, Primary sclerosing cholangitis, European Patent Convention, Research, Communication, Proteomics, Digestate, MD, Doctor of Philosophy, Liver, UPMC, Administration, PSC, Disease, Therapy, Patent, Risk, Patient, Scleroderma, Diagnosis, Biomarker, Clinical trial, CCL24, UC, Pharmaceutical industry

TEL AVIV, Israel, May 09, 2024 (GLOBE NEWSWIRE) -- Chemomab Therapeutics Ltd. (Nasdaq:CMMB), (Chemomab), a clinical-stage biotechnology company developing innovative therapeutics for fibro-inflammatory diseases with high unmet need, today announced financial and operating results for the first quarter ended March 31, 2024, and provided a corporate update.

Key Points: 
  • (Nasdaq:CMMB), (Chemomab), a clinical-stage biotechnology company developing innovative therapeutics for fibro-inflammatory diseases with high unmet need, today announced financial and operating results for the first quarter ended March 31, 2024, and provided a corporate update.
  • In January, 2024, Chemomab reported publication of new proteomics research in the peer-reviewed journal Cells reinforcing the clinical potential of CM-101 in primary sclerosing cholangitis.
  • Research and Development (R&D) Expenses: R&D expenses were $3.1 million for the first quarter of 2024, compared to $6.9 million for the first quarter of 2023.
  • General and Administrative (G&A) Expenses: G&A expenses were $0.9 million for the first quarter of 2024, compared to $2.2 million for the first quarter of 2023.

Capricor Therapeutics to Present Exosome Platform Updates at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

Retrieved on: 
torsdag, maj 9, 2024
Hep G2, Enzyme replacement therapy, Hepatocyte, Cell, Exosome, Partnership, Vaccine, Urea, Therapy, Liver, Gene family, Annual general meeting, Biotechnology, SAN, Arginine

SAN DIEGO, May 09, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX™ exosome platform technology has been selected for an oral presentation at the ASGCT 27th Annual Meeting taking place in Baltimore, Maryland from May 7–11, 2024. The findings highlight a potential exosome-based approach for the treatment of arginase-1 deficiency (ARG1-D), a rare genetic metabolic disease characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells.

Key Points: 
  • The findings highlight a potential exosome-based approach for the treatment of arginase-1 deficiency (ARG1-D), a rare genetic metabolic disease characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells.
  • “The findings from this preclinical study have further characterized our StealthX™ exosome platform and provide a novel approach for exosome-based enzyme-replacement therapies which aim to replace a deficient or absent enzyme,” said Linda Marbán, Ph.D., Capricor’s chief executive officer.
  • “We believe our StealthX™ platform provides an opportunity with potentially broad applications and our data continues to support the concept of exosomes as a suitable delivery vehicle for a variety of payloads.
  • Results showed the Arg1-exosomes were enzymatically active and able to convert arginine into urea in vitro.

Arbor Biotechnologies Presents Data Supporting Clinical Development of ABO-101 and Robust Potential of Platform to Enable Therapeutic Programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

Retrieved on: 
torsdag, maj 9, 2024
Mouse, CRISPR, Impact, Compact, Gene editing, Cell, Artificial intelligence, CGT, PCSK9, IND, CNS, TDP43, PH1, Therapy, Patient, Liver, Gene, Society, ALS, Brain cell, Central nervous system, Messenger, HAO1, NHP, Annual general meeting, Pharmacology, Engineering, Medical device

“This approach has fueled our robust pipeline of genetic medicines which address a range of liver and CNS indications, including PH1 and ALS.

Key Points: 
  • “This approach has fueled our robust pipeline of genetic medicines which address a range of liver and CNS indications, including PH1 and ALS.
  • The data show highly specific targeting of the HAO1 gene in the liver and preservation of genomic integrity upon editing.
  • Together, this strong preclinical data package and the ongoing IND-enabling studies support continued advancement of ABO-101 toward clinical evaluation.
  • Details for the presentations are as follows:
    Oral Presentation Title: Identification and Engineering of ABR-004, a Compact, High-fidelity Nuclease for Therapeutic Gene Editing

Intercept Announces New PBC Data Analyses to be Presented at Digestive Disease Week® 2024 Conference

Retrieved on: 
torsdag, maj 9, 2024
Bezafibrate, Gastrointestinal disease, Learning, Intercept, Abstract, Aspartic acid, Primary biliary cholangitis, Fibrosis, Intercept Pharmaceuticals, Therapy, Senior, Liver, Obeticholic acid, DDW, Hepatology, PBC, Risk, Head, Pharmaceutical industry

MORRISTOWN, N.J., May 09, 2024 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc., a biopharmaceutical company and wholly owned subsidiary of Alfasigma S.p.A. focused on the development and commercialization of novel therapeutics to treat rare and serious liver diseases, today announced five abstracts on primary biliary cholangitis (PBC) will be presented at Digestive Disease Week® (DDW) 2024.

Key Points: 
  • MORRISTOWN, N.J., May 09, 2024 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc., a biopharmaceutical company and wholly owned subsidiary of Alfasigma S.p.A. focused on the development and commercialization of novel therapeutics to treat rare and serious liver diseases, today announced five abstracts on primary biliary cholangitis (PBC) will be presented at Digestive Disease Week® (DDW) 2024.
  • “These data, along with new analyses from our landmark Phase 3 POISE trial, showcase our continued commitment to helping improve the lives of people living with PBC.
  • A full list of sessions at DDW 2024 is available at www.ddw.org .
  • Attendees of DDW can visit Intercept at booths #3741 and #3828 throughout the meeting.

Intellia Therapeutics Announces First Quarter 2024 Financial Results and Highlights Recent Company Progress

Retrieved on: 
torsdag, maj 9, 2024
FDA, Bone marrow, CRISPR, Research, Recode, IND, KLKB1, Medicine, Polyneuropathy, NK, AATD, Corporation, Congress, Brain, TTR, Amyloidosis, Food, Gene editing, Conference, Eye, Corporate social responsibility, BLA, HAE, Therapy, Safety, Tissue, Cystic fibrosis, Cardiomyopathy, ATM, DNA, Intellia Therapeutics, Bank, EAACI, Lung, AAT, Social, ATTR, NTLA, Cell, Disease, Regeneron Pharmaceuticals, Patient, Liver, ESG, Pharmaceutical industry

“Intellia continues to make outstanding progress across our pipeline of in vivo and ex vivo single-dose CRISPR-based therapies.

Key Points: 
  • “Intellia continues to make outstanding progress across our pipeline of in vivo and ex vivo single-dose CRISPR-based therapies.
  • Additionally, Intellia plans to report topline results from the Phase 2 portion in mid-2024 and present full results at a medical meeting in the second half of 2024.
  • Collaboration Revenue: Collaboration revenue was $28.9 million during the first quarter of 2024, compared to $12.6 million during the first quarter of 2023.
  • Net Loss: Net loss was $107.4 million for the first quarter of 2024, compared to $103.1 million during the first quarter of 2023.

Freeline Presents Positive New Data from Phase 1/2 Trial of FLT201, Its Novel Gene Therapy Candidate for Gaucher Disease, in Late-Breaking Oral Presentation at ASGCT 27th Annual Meeting

Retrieved on: 
torsdag, maj 9, 2024
FDA, Bone marrow, Mutation, Cell, Risk, Blood, News, Research, ERT, White blood cell, Glycogen storage disease, SRT, ALT, GBA1, White, European Medicines Agency, Brain, Enzyme replacement therapy, Regenerative Medicine Advanced Therapy, Therapy, Plasma, Patient, Mouse, Safety, Tissue, Glucocerebrosidase, Substantia nigra, Lysosome, Neuropsychological test, AAV, Dementia, PRIME, Fatigue, EMA, Drug development, Bone pain, Gaucher's disease, FACIT, Disease, Liver, Gene, Society, Standard of care, Annual general meeting, Pharmaceutical industry

LONDON, May 09, 2024 (GLOBE NEWSWIRE) --  Freeline Therapeutics today announced new clinical data from its ongoing Phase 1/2 GALILEO-1 trial of FLT201, its adeno-associated virus (AAV) gene therapy candidate for Gaucher disease, showing substantial reductions in glucosylsphinogsine (lyso-Gb1), one of the best predictors of clinical response, in patients with persistently high levels despite years of treatment with currently approved therapies, as well as early signs of clinical improvements in bone marrow burden and fatigue. FLT201 continues to demonstrate a favorable safety and tolerability profile. These data are being showcased in a late-breaking oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place in Baltimore, Maryland.

Key Points: 
  • These data are being showcased in a late-breaking oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place in Baltimore, Maryland.
  • Reductions in lyso-Gb1 levels in the blood are highly correlated with substrate reduction in disease-affected tissues and positive clinical outcomes in Gaucher disease.
  • Currently approved treatments have made a significant difference for people with Gaucher disease, but there is not an existing cure.
  • Both the late-breaking oral presentation and the poster presentation are now available on the News & Events section of Freeline’s website.

89bio Reports First Quarter 2024 Financial Results and Provides Corporate Update

Retrieved on: 
torsdag, maj 9, 2024
MASH, University, Patient, Cirrhosis, Research and development, Liver disease, EASL, Safety, Hypertriglyceridemia, Administration, Journal of Clinical Pathology, Hepatology, Virginia Commonwealth University, PRIME, European Medicines Agency, Liver, Enrollment, EMA, Fibrosis, Division, SAN, Pharmaceutical industry

SAN FRANCISCO, May 09, 2024 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today reported its financial results for the first quarter ended March 31, 2024.

Key Points: 
  • SAN FRANCISCO, May 09, 2024 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today reported its financial results for the first quarter ended March 31, 2024.
  • These key clinical developments, coupled with our agreement for commercial supply, are designed to strategically enhance our readiness for potential commercialization."
  • ENLIGHTEN-Cirrhosis, the second trial in the program, is planned to evaluate patients with compensated cirrhosis and is expected to initiate in the second quarter of 2024.
  • As of March 31, 2024, 89bio had cash, cash equivalents and marketable securities of approximately $562.3 million.