Liver

Vir Biotechnology Announces SOLSTICE Data to be Featured as an Oral Presentation at the European Association for the Study of the Liver (EASL) Congress 2024

Retrieved on: 
tisdag, maj 21, 2024
Biotechnology, Infectious Diseases, Health, Pharmaceutical, Clinical Trials, Hepatology, EASL, AASLD, Safety, Congress, Liver

Vir Biotechnology, Inc. (Nasdaq: VIR) today announced the EASL Congress has changed the Company’s Phase 2 SOLSTICE hepatitis delta presentation from a late-breaker poster to an oral presentation.

Key Points: 
  • Vir Biotechnology, Inc. (Nasdaq: VIR) today announced the EASL Congress has changed the Company’s Phase 2 SOLSTICE hepatitis delta presentation from a late-breaker poster to an oral presentation.
  • The SOLSTICE data will be presented on June 8 during the annual meeting of the European Association for the Study of the Liver, EASL™ Congress 2024, taking place in Milan, Italy, June 5-8.
  • The Company plans to host an investor conference call to discuss the new SOLSTICE data on June 5.
  • The update will include data on participants that have reached 12 and 24 weeks of chronic suppressive treatment.

Liberate Bio Announces Presentation to Update on Advancements on their RAPTOR LNP Platform

Retrieved on: 
torsdag, maj 16, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Patient, Intelligence, Risk, Liver, Disease, Bone marrow, Lipid, Primate, Liberation, Oligonucleotide, RNA, Therapy, Spleen, Vaccine

“We are excited to demonstrate both the establishment of our Rapid Particle Optimizer (RAPTOR™) platform and the first results from our high-throughput screening in the platform at TIDES,” said Walter R. Strapps, Ph.D., Chief Scientific Officer of Liberate.

Key Points: 
  • “We are excited to demonstrate both the establishment of our Rapid Particle Optimizer (RAPTOR™) platform and the first results from our high-throughput screening in the platform at TIDES,” said Walter R. Strapps, Ph.D., Chief Scientific Officer of Liberate.
  • “The presentation demonstrates our ability to rapidly screen a diverse lipid library, designed rationally and by generative artificial intelligence, directly in non-human primates to the genetic medicines community.
  • RAPTOR™ combines the power of high throughput screening with artificial intelligence to efficiently explore vast design spaces covering lipid chemistry, formulation characteristics, and targeting approaches.
  • Our vision at Liberate Bio is to achieve the extraordinary—delivering genetic medicines that transcend liver-based limitations, liberating patients from disease.

Arcturus Therapeutics to Attend Upcoming Investor & Scientific Conferences

Retrieved on: 
onsdag, maj 15, 2024
Infectious Diseases, Biotechnology, Pharmaceutical, Health, RNA, Liver, Infection, ARCT, Vaccine

Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global messenger RNA medicines company focused on the development of infectious disease vaccines and opportunities within liver and respiratory rare diseases, today announced that the Company will participate in the following investor and scientific conferences:

Key Points: 
  • Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global messenger RNA medicines company focused on the development of infectious disease vaccines and opportunities within liver and respiratory rare diseases, today announced that the Company will participate in the following investor and scientific conferences:

GRAIL to Host Capital Markets Day

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måndag, maj 13, 2024
Oncology, Medical Devices, Health, Genetics, Health Technology, Biotechnology, Patient, Illumina, Methylation, Liver, Capital market, Growth, Cancer, Outline, Webcast, MSHS, Management

GRAIL, LLC., a healthcare company whose mission is to detect cancer early when it can be cured, today will host a virtual Capital Markets Day.

Key Points: 
  • GRAIL, LLC., a healthcare company whose mission is to detect cancer early when it can be cured, today will host a virtual Capital Markets Day.
  • Illumina has communicated its goal to finalize divestment terms for GRAIL by the end of the second quarter of 2024.
  • At GRAIL, we are advancing our mission to detect cancer early, when it can be cured, on behalf of patients every day,” said Bob Ragusa, CEO at GRAIL.
  • GRAIL leadership will outline the Company’s core strategy and catalysts for long-term growth, including:
    The live webcast will begin at 10:00 a.m.

Can-Fite: 75 Oncologists and Coordinators from Europe US and Israel Participated in a Conference Aimed at Accelerating Patient Enrolment for the Pivotal Phase 3 Advanced Liver Cancer Study

Retrieved on: 
måndag, maj 13, 2024
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, FDA, Clinical Trials, Patient, Food, European Medicines Agency, Neoplasm, PFS, Trial of the century, Radiology, IDMC, Orphan drug, Inflammation, EMA, Death, Liver, HCC, CANF, American Cancer Society, Liver cancer, Pharmaceutical industry

The participants have been presented with the drug chemical and biological profile and the study protocol as well as study procedures.

Key Points: 
  • The participants have been presented with the drug chemical and biological profile and the study protocol as well as study procedures.
  • The difference in tumor growth or regression defines progression free survival (PFS) and the objective response rate (ORR) to the drug, serving as part of the study objectives.
  • "The conference was very successful and we are very pleased with the enthusiasm of the participants with respect to the drug characteristics.
  • According to the American Cancer Society, liver cancer accounts for more than 700,000 deaths globally each year.

Scribe Therapeutics Presents Data Demonstrating Highly Potent Gene Editing In Vivo, Including Saturated Editing of Hepatocytes in NHPs, by an Engineered XE Genome Editor; Showcases Novel Epigenetic Editing Capabilities at 2024 ASGCT Annual Meeting

Retrieved on: 
fredag, maj 10, 2024
Biotechnology, Pharmaceutical, Genetics, Health, Taste, LNP, Cell, Liver, AAV, Safety, Hepatocyte, Engineering, Gene, CNS, Eye, XE, Society, CRISPR, Epigenetics, Annual general meeting, Vaccine

Scribe Therapeutics Inc. (Scribe), a genetic medicines company unlocking the potential of CRISPR to transform human health, presented data on its X-Editor (XE) and Epigenetic Long-Term X-Repressor (ELXR) technologies at the 27th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place in Baltimore, MD, and virtually.

Key Points: 
  • Scribe Therapeutics Inc. (Scribe), a genetic medicines company unlocking the potential of CRISPR to transform human health, presented data on its X-Editor (XE) and Epigenetic Long-Term X-Repressor (ELXR) technologies at the 27th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place in Baltimore, MD, and virtually.
  • The data described in the company’s oral presentations and poster session showcase the dramatic potency, safety, and specificity of the novel XE genome editing and ELXR epigenetic editing platforms and the company's developments in successfully advancing these technologies into large animal models, including non-human primates.
  • “A major challenge in the field has been to achieve saturating levels of genome editing, at therapeutically relevant doses, with molecules that have better safety and delivery characteristics than CRISPR-Cas9—this has not been possible to date,” said Benjamin Oakes, Ph.D., co-founder and Chief Executive Officer of Scribe.
  • “For the first time, Scribe has demonstrated that the holistic engineering of a wholly unique and highly potent CRISPR enzyme can enable this goal.

Capsida Biotherapeutics to Present Preclinical Data for Parkinson's Disease Associated with GBA Mutations Showing High Levels of GCase Enzyme Supplementation Following IV Administration

Retrieved on: 
fredag, maj 10, 2024
Traffic barrier, Patient, Cell, PD, Parkinson's disease, Baltimore Convention Center, Liver, Dorsal root ganglion, Disease, DSM-IV codes, Gene, Neuron, Cerebrospinal fluid, Brain, Society, Annual general meeting, GBA, Potential

THOUSAND OAKS, Calif., May 10, 2024 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") today announced new preclinical data that demonstrate the potential of its next-generation intravenously (IV) administered gene therapy candidate, CAP-003, to safely and effectively treat Parkinson's disease (PD) associated with GBA mutations (PD-GBA). Collectively, the findings strongly support the best-in-class profile of CAP-003 and Capsida's plans to advance the therapy into clinical trials in the first half of 2025.

Key Points: 
  • Mutations in GBA, the gene expressing the GCase enzyme, are the most common genetic risk factor for PD.
  • Other treatments for PD-GBA have been limited by their inability to cross the blood-brain barrier and supplement GCase enzyme activity in sufficient quantities to impact the disease.
  • Capsida's wholly owned novel gene therapy offers the potential to supplement the GCase enzyme with a single IV infusion, enabling long-term disease modification and substantially slowing disease progression.
  • "Our novel wholly owned PD-GBA gene therapy demonstrates the potential to provide long-term significant decrease in disease progression safely with a single IV infusion," said Capsida CEO Peter Anastasiou.

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: 
fredag, maj 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.

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.