Hepatocyte

Wave Life Sciences to Host Analyst and Investor Virtual Event, “Towards the Clinic: Spotlight on RNA Editing for AATD,” on September 28, 2022

Retrieved on: 
Friday, September 16, 2022
Critical Care Medicine (journal), Enzyme, VP, MicroRNA, SERPINA1, Human, ADAR, Q&A, MD, Adenosine, Oligonucleotide, Inosine, Wave, Patient, Chicago (franchise), List of life sciences, Adenine, MBA, PRISM, Genetic disorder, Liver, Degenerative disease, Doctor of Philosophy, Drug development, Aimer, Guanine, RNA, Lists of diseases, GLOBE, Hepatocyte, University, Biology, Webcast, Disease, Chemistry, Outline, Pharmaceutical industry, AATD, Medicine

A live webcast of the event will be available on the investor relations page of theWave Life Sciencescorporate website at http://ir.wavelifesciences.com .

Key Points: 
  • A live webcast of the event will be available on the investor relations page of theWave Life Sciencescorporate website at http://ir.wavelifesciences.com .
  • Following the live event, an archived version of the webcast will be available on the Wave Life Sciences website.
  • Waves A-to-I RNA base editing oligonucleotides (AIMers) are designed to recruit these endogenous ADAR enzymes to direct efficient and highly specific editing of RNA transcripts.
  • Wave Life Sciences (Nasdaq: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases.

Castle Creek Biosciences Announces Publication of Preclinical Study for Potential In Vivo Gene Therapy in Nature Communications

Retrieved on: 
Tuesday, August 30, 2022
Paragon, Connective tissue, Mayo Clinic, Liver failure, Cirrhosis, HCC, Research, Hepatocyte, Lists of diseases, Liver, Doctor of Philosophy, Tyrosine, Disease, Hope, Hepatocellular carcinoma, Skin, FAH, Stress (linguistics), Genome, Nature Communications, Liver disease, MD, Fibrosis, Patient, Biology, St. John's Hospital (Maplewood, Minnesota), Regenerative medicine, Mayo, Clinical trial, Tyrosinemia, Metabolism, Vaccine, Pharmaceutical industry, Medical imaging

EXTON, Pa., Aug. 30, 2022 /PRNewswire/ -- Castle Creek Biosciences announced publication of a preclinical study that found that its experimental in vivo gene therapy treatment resolved biochemical and hepatic histological markers of hereditary tyrosinemia type-1 (HT1) in a large animal model of the disease. The findings appear in the August 25 edition of Nature Communications.

Key Points: 
  • EXTON, Pa., Aug. 30, 2022 /PRNewswire/ --Castle Creek Biosciences announced publication of a preclinical study that found that its experimental in vivo gene therapy treatment resolved biochemical and hepatic histological markers of hereditary tyrosinemia type-1 (HT1) in a large animal model of the disease.
  • Castle Creek Biosciences in vivo gene therapy platform featured in Nature Communications.
  • The direct in vivo delivery of a single intraportal dose of the vector carrying the human FAH gene normalized liver function 78-98 days post-treatment, according to the published study.
  • Castle Creek Biosciences is a late-stage cell and gene therapy company developing treatments for skin, connective tissue and metabolic diseases.

Arbutus Biopharma Announces Issuance of a New Key U.S. Patent Related to AB-729

Retrieved on: 
Tuesday, August 30, 2022
RNA interference, Liver cancer, CFBV, COVID-19, N-Acetylgalactosamine, HBV, RNA, Securities Exchange Act of 1934, Cirrhosis, Program, Membrane protein, Hepatocyte, Hepatitis B virus, Securities Act of 1933, Arbutus, Patent, Virology, Vaccine, Death, USPTO, Coronavirus, Risk, Hepatitis B, World Health Organization, Antigen, Goal, Degenerative disease, Severe acute respiratory syndrome coronavirus 2, GLOBE, PD, Nasdaq, Immune system, Therapy, NA, Conditional sentence, World, Drug discovery, Annual report, Forward-looking statement, Patient, Pharmaceutical industry

11,427,823, which provides composition of matter patent protection for Arbutus AB-729 RNAi therapeutic product.

Key Points: 
  • 11,427,823, which provides composition of matter patent protection for Arbutus AB-729 RNAi therapeutic product.
  • The patent is expected to provide Arbutus with exclusivity for AB-729 out to at least April 2038.
  • Furthermore, AB-729 has shown achievement of virologic control in five cHBV patients after discontinuing treatment with AB-729 and nucleos(t)ide analog (NA)-therapy.
  • Arbutus Biopharma Corporation (Nasdaq: ABUS) is a clinical-stage biopharmaceutical company leveraging its extensive virology expertise to develop novel therapeutics that target specific viral diseases.

Arbor Biotechnologies Enters into Agreement with Acuitas Therapeutics for Lipid Nanoparticle Delivery System for Use in Rare Liver Diseases

Retrieved on: 
Thursday, August 25, 2022
Partnership, COVID-19, CEO, Technology, Cancer, Hepatocyte, Institute of Liver and Biliary Sciences, Liver, Therapy, Gene editing, Vaccine, Toxicity, Disease, Lists of diseases, Rabies, Pfizer–BioNTech COVID-19 vaccine, GLOBE, CRISPR, LNP, Company, Biotechnology, Paul Reynolds (commentator), Tuberculosis, Industry, Malaria, CNS, HIV/AIDS, Patient, Pharmaceutical industry

We are looking forward to working with Acuitas, a leading global developer of clinically-validated LNP technology, said Devyn Smith, Ph.D., CEO, Arbor Biotechnologies.

Key Points: 
  • We are looking forward to working with Acuitas, a leading global developer of clinically-validated LNP technology, said Devyn Smith, Ph.D., CEO, Arbor Biotechnologies.
  • Founded in February 2009, Vancouver-based Acuitas Therapeutics ( www.acuitastx.com ) is a private biotechnology company that specializes in the development of delivery systems for nucleic acid therapeutics based on lipid nanoparticles.
  • Acuitas Therapeutics has agreements in place with several partners to use its proprietary lipid nanotechnology in the development of COVID-19 vaccines.
  • The Acuitas team is currently working on therapeutics focused on addressing cancer, HIV/AIDS, tuberculosis, malaria, rabies, and other serious diseases.

Bluejay Therapeutics raises $41 million in Series B round to drive clinical trials in chronic hepatitis

Retrieved on: 
Tuesday, August 16, 2022
Lazarus, Immunoglobulin G, Hepatocyte, CEO, GLOBE, Blue, Growth, Leadership, Investment, HBV, IND, Board, Patient, Therapy, Infection, SAN, CHBV, Series, Hepatitis B, Institutional review board, HBsAg, Immunology, Blue jay, Disease, Pharmaceutical industry

SAN MATEO, Calif., Aug. 16, 2022 (GLOBE NEWSWIRE) -- Bluejay Therapeutics announced today that it has closed a $41 million Series B round of finance led by Arkin Bio Ventures.

Key Points: 
  • SAN MATEO, Calif., Aug. 16, 2022 (GLOBE NEWSWIRE) -- Bluejay Therapeutics announced today that it has closed a $41 million Series B round of finance led by Arkin Bio Ventures.
  • It will also fund development of BJT-574, an orally bioavailable small molecule HBsAg inhibitor, into first-in-human clinical trials.
  • The companys first target indication is chronic Hepatitis B, which remains a worldwide prevalent disease with urgent unmet medical need.
  • The company believes that by reducing hepatitis B surface antigen and restoring adaptive immunity a functional cure could be achieved for patients.

MediciNova Announces MN-001 (tipelukast) Abstract regarding Improvement of Serum Lipid Panel in Type 2 Diabetes and NAFLD Patients Accepted for Presentation at the IDF 2022 Congress, the Annual Meeting of the International Diabetes Federation

Retrieved on: 
Tuesday, August 9, 2022
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This subgroup analysis was conducted based on in-vitro research findings that MN-001 down regulated CD36 mRNA and upregulated ABCG1 mRNA.

Key Points: 
  • This subgroup analysis was conducted based on in-vitro research findings that MN-001 down regulated CD36 mRNA and upregulated ABCG1 mRNA.
  • NAFLD is considered the hepatic manifestation of metabolic syndrome; studies have reported that 50% of patients with metabolic syndrome also have NAFLD.
  • The presence of dyslipidemia (hypercholesterolemia, hypertriglyceridemia, or both) is reported in 20 - 80% of NAFLD cases.
  • Undue reliance should not be placed on these forward-looking statements, which speak only as of the date hereof.

Generation Bio Reports Business Highlights and Second Quarter 2022 Financial Results

Retrieved on: 
Thursday, August 4, 2022
Rubius, U.S. Securities and Exchange Commission, Hepatocyte, Private Securities Litigation Reform Act, GLOBE, R, Board, Security (finance), Research, Chris Appelhans, Good manufacturing practice, DNA, Therapy, COVID-19, D, Form 10-K, GMP, G&A, Board of directors, Generation, Retina, Annual report, RES, Vaccine, Risk management

CAMBRIDGE, Mass., Aug. 04, 2022 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported business highlights and second quarter 2022 financial results.

Key Points: 
  • CAMBRIDGE, Mass., Aug. 04, 2022 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported business highlights and second quarter 2022 financial results.
  • R&D Expenses:Research and development (R&D) expenses were$28.4millionfor the quarter ended June 30, 2022, compared to $22.7 million for the quarter ended June 30, 2021.
  • G&A Expenses:General and administrative (G&A) expenses were$10.1millionfor the quarter ended June 30, 2022, compared to $8.2 million for the quarter ended June 30, 2021.
  • Generation Bio is innovating genetic medicines to provide durable, redosable treatments for people living with rare and prevalent diseases.

Sana Biotechnology Reports Second Quarter 2022 Financial Results and Business Updates

Retrieved on: 
Thursday, August 4, 2022
Serum, Survival, Risk, Social disruption, RPE, GLOBE, HIP, Cancer, Research, Degenerative disease, Patient, Biotechnology, Research and development, State Administrative Expenses, Development, Immunosuppression, Terminology, Disease, Primate, Hepatocyte, Gene, Technology, Potency (pharmacology), CD22, Contingency (philosophy), Safety, IND World's Fair Line, BCMA, Income, Retinal pigment epithelium, Cell, COVID-19, Drug development, ISSCR, AACR, SANA, Organization, ADA, IND, Company, NASDAQ, GAAP, Retinal, Administration, Information technology, Mobile phone, Pharmaceutical industry, Vaccine, Cryptocurrency, Online gambling, Reinsurance

SEATTLE, Aug. 04, 2022 (GLOBE NEWSWIRE) -- Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, today reported financial results and business highlights for the second quarter 2022.

Key Points: 
  • We are pleased with the continued progress this quarter both across our platforms and with our product candidates.
  • Sana also presented data showing that HIP allogeneic regulatory T cells function and are able to evade immune detection in preclinical models.
  • These data provide a path for a potential safety strategy as well as validation of the mechanism of immune protection.
  • The Company has based these forward-looking statements largely on its current expectations, estimates, forecasts and projections about future events and financial trends that it believes may affect its financial condition, results of operations, business strategy and financial needs.

Induced Pluripotent Stem Cell (iPSC) Global Market Report 2022: Featuring Key Players Thermo Fisher Scientific, Bristol-Myers Squibb, Takara Bio & Others - ResearchAndMarkets.com

Retrieved on: 
Wednesday, July 27, 2022
Biotechnology, Health, Stem Cells, Association, Diabetes, Time, Fibroblast, Population, Tissue, Conditional sentence, IPSC, Cell potency, Partnership, Stem cell, Risk, American Association, Adult, COVID-19, Application, Degenerative disease, Cancer, Neoplasm, CAGR, Patient, Stroke, Death, Growth, Hepatocyte, Prevalence, Child, Mobile phone, Pharmaceutical industry, Keratinocyte

The "Induced Pluripotent Stem Cell (iPSC) Global Market Report 2022, By Derived Cell Type, By Application, By End-User" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Induced Pluripotent Stem Cell (iPSC) Global Market Report 2022, By Derived Cell Type, By Application, By End-User" report has been added to ResearchAndMarkets.com's offering.
  • The global induced pluripotent stem cell (iPSC) market is expected to grow from $ 2431.2 million in 2021 to $ 2640.80 million in 2022 at a compound annual growth rate (CAGR) of 8.6%.
  • Increase in the prevalence of chronic disorders is one of the major factors that is driving the growth of Induced pluripotent stem cell market.
  • The potential risk of tumour is one of the major restraints on the growth of Induced pluripotent stem cell market.

MediciNova Announces Initiation of a Phase 2 Trial of MN-001 (tipelukast) in NAFLD with Type 2 Diabetes Mellitus and Hypertriglyceridemia

Retrieved on: 
Tuesday, July 26, 2022
Proton, Patient, Male, IPF, Dyslipidemia, Multiple sclerosis, GLOBE, LOXL2, PDE, Malignancy, Hypertriglyceridemia, Glioblastoma, U.S. Securities and Exchange Commission, Amyotrophic lateral sclerosis, Female, Doctor of Philosophy, T2DM, Hypercholesterolemia, Neurodegeneration, Code, Cardiovascular disease, Intellectual property, Form 8-K, Incidence, Private Securities Litigation Reform Act, NAFLD, NDA, Chronic kidney disease, Fibrosis, Risk, Failure, Safety, Arachidonic acid, DCM, Gene, MCP-1, Fasting, Inflammation, MS, Metabolic syndrome, ALS, FDA, MD, Security (finance), Tokyo Stock Exchange, Nasdaq, Annual report, Week, Hepatocyte, Idiopathic pulmonary fibrosis, Non-alcoholic fatty liver disease, Form 10-K, CCR2, Prevalence, Element, Pharmaceutical industry

Kazuko Matsuda, MD, PhD, Chief Medical Officer of MediciNova, Inc., commented, "We are very pleased that this Phase 2 trial evaluating MN-001 has commenced.

Key Points: 
  • Kazuko Matsuda, MD, PhD, Chief Medical Officer of MediciNova, Inc., commented, "We are very pleased that this Phase 2 trial evaluating MN-001 has commenced.
  • MN-001 appears to reduce serum lipid profiles in patients with a dual diagnosis of NAFLD and T2DM/prediabetes with dyslipidemia.
  • This is a Phase 2, multi-center, randomized, double-blind, placebo-controlled clinical trial to evaluate the efficacy and safety of MN-001 (tipelukast) in patients with NAFLD, T2DM and hypertriglyceridemia.
  • The design of the Phase 2 clinical trial includes the following elements:
    Approximately 40 male and female patients, ages 21 to 75 years, in the U.S.