Hepatocyte

Sirnaomics will Present its Innovative Dual-Targeted GalNAc muRNA Programs in 2024 OPT Conference

Retrieved on: 
Tuesday, March 12, 2024
Gene, Congress, Oligonucleotide, Patient, RNA, Messenger, N-Acetylgalactosamine, Hepatocyte, Therapy, Múrna, RNA interference, Liver

Based on Sirnaomics' continued progress in targeting single genes with GalAheadTM mxRNA, Company is now pioneering GalAheadTM muRNA, a proprietary RNAi-based platform that allows for simultaneous downregulating of multiple genes.

Key Points: 
  • Based on Sirnaomics' continued progress in targeting single genes with GalAheadTM mxRNA, Company is now pioneering GalAheadTM muRNA, a proprietary RNAi-based platform that allows for simultaneous downregulating of multiple genes.
  • GalAheadTM muRNA, alike mxRNA employs GalNAc ligands, a clinically validated concept for RNAi trigger delivery to liver hepatocytes providing subsequent treatment of liver associated diseases.
  • The cleavage points allow for the muRNA after cellular delivery to break into two individual RNAi-triggers in the endo-lysosomal pathway.
  • In a different scenario, muRNA can simultaneously address two or more non-associated indications where patient populations have a considerable overlap.

Generation Bio Reports Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, March 6, 2024
Therapy, Research, Haemophilia, DSM-IV codes, HSC, Health, Thalassemia, Sickle cell disease, G&A, Hepatocyte, DNA, Mouse, Vaccine

CAMBRIDGE, Mass., March 06, 2024 (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 fourth quarter and full year 2023 financial results.

Key Points: 
  • (Nasdaq:GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported business highlights and fourth quarter and full year 2023 financial results.
  • “Late last year we announced important advances in our cell-targeted lipid nanoparticle and immune-quiet DNA platforms that we are now translating to our portfolio strategy,” said Geoff McDonough, M.D., chief executive officer of Generation Bio.
  • We are also continuing to optimize iqDNA to progress our hemophilia A program and to explore therapeutic applications in cell types beyond hepatocytes.
  • In 2024, the company intends to optimize iqDNA for application in hepatocytes and T cells with a focus on improving potency.

Ribo partners with Boehringer Ingelheim to develop new treatments for people with liver diseases

Retrieved on: 
Wednesday, January 3, 2024
RNA, RNA interference, Liver, Partnership, NASH, Boehringer Ingelheim, Cirrhosis, Hepatocyte, Small RNA, Boehringer, Research, Messenger, USD, DSM-IV codes, CRM, Liver failure, Liver cancer, Patient, Therapy, Senior, Pharmaceutical industry

INGELHEIM, Germany, MÖLNDAL, Sweden and KUNSHAN, China, Jan. 3, 2024 /PRNewswire/ -- Suzhou Ribo Life Science Co., Ltd. and Ribocure AB (Ribo) today announced a collaboration with Boehringer Ingelheim to develop novel treatments for nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH).

Key Points: 
  • INGELHEIM, Germany, MÖLNDAL, Sweden and KUNSHAN, China, Jan. 3, 2024 /PRNewswire/ -- Suzhou Ribo Life Science Co., Ltd. and Ribocure AB (Ribo) today announced a collaboration with Boehringer Ingelheim to develop novel treatments for nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH).
  • More than 440 million people worldwide are estimated to live with NASH, an inflammatory liver disease that is caused by accumulation of fat in the liver.
  • Over time, NASH causes scar tissue formation, which in many cases leads to liver cirrhosis and related serious complications, including liver failure or liver cancer.
  • We are very pleased about the opportunity to work with Boehringer Ingelheim to develop new solutions for people living with NASH."

Ribo partners with Boehringer Ingelheim to develop new treatments for people with liver diseases

Retrieved on: 
Wednesday, January 3, 2024
RNA, RNA interference, Liver, Partnership, NASH, Boehringer Ingelheim, Cirrhosis, Hepatocyte, Small RNA, Boehringer, Research, Messenger, USD, DSM-IV codes, CRM, Liver failure, Liver cancer, Patient, Therapy, Senior, Pharmaceutical industry

INGELHEIM, Germany, MÖLNDAL, Sweden and KUNSHAN, China, Jan. 3, 2024 /PRNewswire/ -- Suzhou Ribo Life Science Co., Ltd. and Ribocure AB (Ribo) today announced a collaboration with Boehringer Ingelheim to develop novel treatments for nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH).

Key Points: 
  • INGELHEIM, Germany, MÖLNDAL, Sweden and KUNSHAN, China, Jan. 3, 2024 /PRNewswire/ -- Suzhou Ribo Life Science Co., Ltd. and Ribocure AB (Ribo) today announced a collaboration with Boehringer Ingelheim to develop novel treatments for nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH).
  • More than 440 million people worldwide are estimated to live with NASH, an inflammatory liver disease that is caused by accumulation of fat in the liver.
  • Over time, NASH causes scar tissue formation, which in many cases leads to liver cirrhosis and related serious complications, including liver failure or liver cancer.
  • We are very pleased about the opportunity to work with Boehringer Ingelheim to develop new solutions for people living with NASH."

Omega Therapeutics Showcases Bidirectional and Multiplexed Epigenomic Control of Gene Expression in Preclinical Models of Liver Inflammation and Fibrosis

Retrieved on: 
Monday, November 13, 2023
Therapy, CXCL10, CXCL11, Cirrhosis, AASLD, ECS, Mouse, CXCL9, Liver, Gene expression, Messenger, Hepatocyte, FRG, Chemokine, Cell, Inflammation, Fibrosis, Vaccine, Boston

CAMBRIDGE, Mass., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Omega Therapeutics, Inc. (Nasdaq: OMGA) (“Omega”), a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines, today announced the presentation of new preclinical data from two different programs that demonstrated sustained upregulation of gene expression and coordinated pre-transcriptional downregulation of multiple genes in models of liver fibrosis and inflammation, respectively, at the American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting® 2023, taking place in Boston, Massachusetts, November 10 – 14.

Key Points: 
  • However, to extend their reach, we need to bidirectionally control the expression of multiple genes simultaneously,” said Thomas McCauley, Ph.D., Chief Scientific Officer of Omega Therapeutics.
  • “We believe that these new data demonstrate the power of our programmable epigenomic mRNA development candidates to control gene expression with unmatched flexibility.
  • To our knowledge, these are the first results to show how site-specific epigenomic modulation can durably upregulate the expression of a master liver regeneration gene.
  • EC-mediated induction of HNF4α expression in vivo in a mouse model of liver fibrosis led to decreased collagen deposition, a key marker of fibrosis.

Precision BioSciences Announces Approval of First Clinical Trial Application of ARCUS Gene Insertion Program by Partner iECURE

Retrieved on: 
Wednesday, December 13, 2023
Research, Infectious Diseases, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, General Health, Science, Precision BioSciences, Nerve, Gene editing, Ornithine transcarbamylase deficiency, HDR, Gene, Clinical trial, Coma, IND, Liver, Disease, Risk, Patient, Cell, ESGCT, Blood, Hepatocyte, Primate, CRISPR, Ammonia, Death, Neurology, Congress, CTA, OTC, Precision, Metabolic disorder, Vaccine

“The acceptance of iECURE’s Clinical Trial Application marks an important milestone for patients with OTC deficiency and for both the iECURE and Precision BioSciences teams.

Key Points: 
  • “The acceptance of iECURE’s Clinical Trial Application marks an important milestone for patients with OTC deficiency and for both the iECURE and Precision BioSciences teams.
  • This is the first ARCUS in vivo gene editing program to progress into the clinic,” said Michael Amoroso, Chief Executive Officer of Precision Biosciences.
  • A 5% threshold of enzymatic activity has the potential for clinical benefit as previously identified in Annals of Clinical and Translational Neurology .
  • “ECUR-506 is the first of several partnered and wholly owned gene editing programs leveraging ARCUS in differentiated indications and we look forward to initial clinical data from this program.”

Tune Therapeutics Presents First Data Supporting TUNE-401: a First-in-Class Epigenetic Silencer for Hepatitis B

Retrieved on: 
Wednesday, December 6, 2023
Research, Infectious Diseases, Genetics, Clinical Trials, Other Health, Biotechnology, General Health, Health, Science, Other Science, DNA methylation, Chromosome, DNA, Therapy, Hepatitis B virus, Liver, Tune, Mouse, HBV, Patient, TEMPO, Chimera, Hepatocyte, PCSK9, Genome, FRG, Vaccine, Dietary supplement, Medical device, CccDNA

Leading epigenome editing company Tune Therapeutics presented data in support of its chronic Hepatitis B Virus (HBV) program, demonstrating the ability to durably silence essential mechanisms of viral replication and persistence across a range of model systems.

Key Points: 
  • Leading epigenome editing company Tune Therapeutics presented data in support of its chronic Hepatitis B Virus (HBV) program, demonstrating the ability to durably silence essential mechanisms of viral replication and persistence across a range of model systems.
  • Importantly, the TEMPO platform does this via epigenetic processes, and without cutting, damaging, or altering genomic DNA sequences in any way.
  • “Through the richness of these data sets, we found multiple repression candidates that worked well in both contexts.
  • As such, they represent the gold standard for assessing the dose and efficacy for liver-directed therapeutics ahead of human clinical trials.

HOOKIPA Pharma Reports Third Quarter 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Thursday, November 9, 2023
Hepatocyte, Prostate-specific antigen, Research, Trial of the century, IND, PSA, Infection, Hepatitis, Pembrolizumab, Head, HIV, Liver, KRAS, Arenavirus, Prostate cancer, Gilead Sciences, Journal, CPI, Development, Gilead, Lung, PAP, Oncology, Serum, Safety, European Society of Gynaecological Oncology, Cancer, Patient, Administration, HBV, The Journal of Infectious Diseases, Investigational New Drug, Pharmaceutical industry, Clothing, Vaccine, Reinsurance

With these encouraging data, we are busy finalizing plans for the randomized trial expected to begin next year,” said Joern Aldag, Chief Executive Officer at HOOKIPA Pharma.

Key Points: 
  • With these encouraging data, we are busy finalizing plans for the randomized trial expected to begin next year,” said Joern Aldag, Chief Executive Officer at HOOKIPA Pharma.
  • Enrollment continued in the ongoing Phase 1/2 study ( NCT05553639 ) of HB-300 for the treatment of advanced prostate cancer.
  • HB-300 is an arenaviral immunotherapy that targets two well-defined self-antigens of prostate cancer, prostatic acid phosphatase (PAP) and prostate-specific antigen (PSA).
  • The decrease was primarily attributable to cash used in operating activities, partly offset by funds resulting from the follow-on financing in June 2023.

Galectin Therapeutics to Share Five Scientific Presentations at The Liver Meeting™ 2023, Hosted by the AASLD

Retrieved on: 
Friday, November 3, 2023
Hepatocyte, Hynes Convention Center, American Association for the Study of Liver Diseases, Therapy, Hepatology, Liver, Pharmacokinetics, Sarcopenia, Safety, GALT, Glomerular filtration rate, GA 2, AM, Abstract (summary), Obesity, Indiana University School of Medicine, Cirrhosis, Data monitoring committee, Disease, Hepatic hydrothorax, Medical school, Indiana University, SC 4, K–Ca dating, Non-alcoholic fatty liver disease, AASLD, Dietary supplement, Pharmaceutical industry, Flour, Patient

NORCROSS, Ga., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of therapeutics that target galectin-3, today announced that it will share five scientific presentations at The Liver Meeting™ 2023, hosted by the American Association for the Study of Liver Diseases (AASLD).

Key Points: 
  • NORCROSS, Ga., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of therapeutics that target galectin-3, today announced that it will share five scientific presentations at The Liver Meeting™ 2023, hosted by the American Association for the Study of Liver Diseases (AASLD).
  • The Liver Meeting™ will be held November 10-14, 2023, in Boston, Mass.
  • “This year again we are happy to share some of the progress we have made in our global program with belapectin, our galectin-3 inhibitor.
  • We are also proud to bring additional knowledge to the field regarding liver cirrhosis, a disease that dramatically affects the lives of our patients and has been neglected for far too long,” said Pol Boudes, M.D., Chief Medical Officer of Galectin Therapeutics.

Prime Medicine Presents First-ever Prime Editing Data in Non-human Primates Demonstrating Highly Efficient Ability of Prime Editors to Precisely Correct Disease-causing Mutation of GSD1b

Retrieved on: 
Friday, October 27, 2023
Well, Gene editing, NHP, RNA, Biotechnology, Congress, SLC37A4, Liver, Cell, Allele, Hypoglycemia, Caregiver, Disease, Gene, Messenger, LNP, ESGCT, Animal, European Society of Gene and Cell Therapy, Patient, Mutation, Vaccine, Marketing, Hepatocyte

CAMBRIDGE, Mass., Oct. 27, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology Company committed to delivering a new class of differentiated, one-time curative genetic therapies, today reported new preclinical data demonstrating the ability of liver-targeted Prime Editors to efficiently and precisely correct one of the most prevalent disease-causing mutations of glycogen storage disease 1b (GSD1b) in non-human primates (NHP) and mouse models. The data were presented today at the European Society of Gene and Cell Therapy (ESGCT) 2023 Congress in Brussels, Belgium.

Key Points: 
  • The data were presented today at the European Society of Gene and Cell Therapy (ESGCT) 2023 Congress in Brussels, Belgium.
  • “The data presented today are highly encouraging, both for patients and caregivers impacted by GSD1b, as well as for Prime Medicine and the field of gene editing,” said Jeremy Duffield, M.D., Ph.D., Chief Scientific Officer of Prime Medicine.
  • “These data are the first Prime Editing data in NHPs and provide further proof-of-concept for our Prime Editing approach to potentially address a wide range of diseases, in this case, by targeting a specific gene with a liver-directed LNP.
  • In today’s presentation at ESGCT, Prime Medicine highlighted data from in vivo rodent and NHP studies with its Prime Editor targeting the p.L348fs mutation.