LDLR

Verve Therapeutics Announces Pipeline Progress and Reports First Quarter 2024 Financial Results

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Mercredi, mai 8, 2024
LDLR, Verve, Safety, Pharmacokinetics, ASCVD, LNP, Eli Lilly, Moyamoya disease, N-Acetylgalactosamine, RNA, Aortic stenosis, Stroke, Cell, Conference, Research, Webcast, PCSK9, IND, Administration, Potential, Research and development, Patient, Liver, Gene, CAD, Society, Medicine, ANGPTL3, Lilly, Cardiovascular disease, CTAS, Therapy, Asialoglycoprotein receptor, G&A, LPA, Pharmaceutical industry

BOSTON, May 08, 2024 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today reported pipeline updates and financial results for the quarter ended March 31, 2024.

Key Points: 
  • Cash Position: Verve ended the first quarter of 2024 with $606.4 million in cash, cash equivalents, and marketable securities.
  • Collaboration Revenue: Collaboration revenue was $5.7 million for the first quarter of 2024, compared to $1.4 million for the first quarter of 2023.
  • General & Administrative (G&A) Expenses: G&A expenses were $14.2 million for the first quarter of 2024, compared to $12.6 million for the first quarter of 2023.
  • Stock-based compensation expense included in G&A expenses was $4.7 million and $3.5 million for the first quarter of 2024 and 2023, respectively.

Verve Therapeutics Announces Dosing of First Patient in Heart-2 Phase 1b Clinical Trial Evaluating VERVE-102

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Mardi, mai 7, 2024
LDLR, Patient, Clinical trial, Risk, Safety, Woman, Pharmacokinetics, Blood, ASCVD, Gene, Moyamoya disease, Gene editing, RNA, PCSK9, Life, Ageing, Liver, CAD, Cardiovascular disease, Therapy, Asialoglycoprotein receptor, DSM-IV codes, Apolipoprotein, Sudden death, Pharmaceutical industry

BOSTON, May 07, 2024 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today announced that the first patient has been dosed with VERVE-102 in the Heart-2 Phase 1b clinical trial. The Heart-2 trial is enrolling adult patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD), two patient populations who require deep reductions of low-density lipoprotein cholesterol (LDL-C) levels in the blood for an extended period of time. Patients living with HeFH have an inherited disorder characterized by elevated blood levels of LDL-C starting early in life. Patients living with premature CAD experience cholesterol-driven blockage of coronary arteries early in life and are at high risk of further complications. A lack of durable control of LDL-C levels in both HeFH and premature CAD patients carries high lifetime risks for cardiovascular events, including heart attack and sudden death.

Key Points: 
  • Patients living with HeFH have an inherited disorder characterized by elevated blood levels of LDL-C starting early in life.
  • Patients living with premature CAD experience cholesterol-driven blockage of coronary arteries early in life and are at high risk of further complications.
  • “Dosing the first patient in the Heart-2 Phase 1b clinical trial for VERVE-102 is an important step in the continued progress of our pipeline,” said Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve Therapeutics.
  • Clinical Trial Applications for the Heart-2 trial have been cleared in Canada and the United Kingdom.

Epic Bio Presents New Data Highlighting Potential of EPI-321 for FSHD and Epigenetic Editing Platform Leadership at ASGCT Annual Meeting

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Mardi, avril 23, 2024
LDLR, Cas9, Facioscapulohumeral muscular dystrophy, CRISPR, Safety, RNA, Cas12a, Cell, Translation, Patient, Gene, FSHD, Society, LDL, Genome, VPR, Annual general meeting, SAN, Vaccine

Across three oral presentations and three poster presentations, Epic Bio will present new data demonstrating the differentiation of the company’s epigenetic editing platform and the promise of lead candidate EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

Key Points: 
  • Across three oral presentations and three poster presentations, Epic Bio will present new data demonstrating the differentiation of the company’s epigenetic editing platform and the promise of lead candidate EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
  • A second study evaluated the use of proprietary epigenetic activators to boost transcription of the LDL Receptor (LDLR) gene.
  • As a leader in the field of epigenetic editing, Epic Bio has done pioneering work to craft a comprehensive off-target assessment platform to ensure the safety of epigenetic editing therapies for human testing.
  • A third oral presentation at ASGCT spotlights a novel, freely available tool recently developed by Epic Bio to assist researchers who wish to use the hypercompact dCasMINI protein for epigenetic editing.

Vect-Horus Publication in Pharmaceutics Outlines How Vectors Can Improve Delivery of Therapeutics to Organs and Tumors

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Lundi, avril 22, 2024
LDLR, Small RNA, Cancer, Cell, Central nervous system disease, Tissue, MDPI, RNA, GBM, CNS, PDAC, Novo Nordisk, Family, Peptide, Neoplasm, Gene expression, Glioblastoma, Biotechnology, Ionis, Vaccine

Because the LDLR is differentially expressed in organs and is overexpressed in many cancers including glioblastoma multiforme (GBM) and pancreatic ductal adenocarcinoma (PDAC), the results open new opportunities for delivery to different organs or tumors.

Key Points: 
  • Because the LDLR is differentially expressed in organs and is overexpressed in many cancers including glioblastoma multiforme (GBM) and pancreatic ductal adenocarcinoma (PDAC), the results open new opportunities for delivery to different organs or tumors.
  • A vector belonging to this family of LDLR-targeting peptides is applied as a theragnostic agent for the diagnostic of GBM and PDAC.
  • “These results reinforce the potential of vectors to significantly improve the transport and delivery of therapeutic and imaging agents to improve the diagnostic and treatment of cancer and CNS disorders,” said Alexandre Tokay co-founder and CEO of Vect-Horus.
  • The study identified and optimized a family of peptide-based vectors that target the LDLR.

Verve Therapeutics Announces Updates on its PCSK9 Program

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Mardi, avril 2, 2024
LDLR, Cardiovascular disease, Gene editing, PCSK9, ALT, Patient, RNA, Health care, Health Canada, IND, N-Acetylgalactosamine, MHRA, Asialoglycoprotein receptor, Clinical trial, CTAS, Therapy, Bleeding, Safety, Moyamoya disease, DSMB, FDA, Liver, ASCVD, LNP, Medsafe, Hypercholesterolemia, Food, Pharmaceutical industry

BOSTON, April 02, 2024 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today announced updates from the Heart-1 Phase 1b clinical trial of VERVE-101 and clearance of its Clinical Trial Applications (CTAs) by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and Health Canada for VERVE-102, with the Heart-2 Phase 1b clinical trial expected to initiate in the second quarter of this year.

Key Points: 
  • Six participants have been dosed at 0.45 mg/kg of VERVE-101, with a total of 13 participants dosed in the study.
  • Verve is conducting an investigation into the laboratory abnormalities and based on those results, expects to work with regulatory authorities to define a path forward for VERVE-101.
  • Verve is now prioritizing the development of VERVE-102 and the initiation of the Heart-2 clinical trial.
  • VERVE-102 uses the same base editor and guide RNA for PCSK9 but a different lipid nanoparticle (LNP) delivery system than VERVE-101.

Verve Therapeutics Provides Pipeline Progress and Reports Fourth Quarter and Full Year 2023 Financial Results

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Mardi, février 27, 2024
Beam Therapeutics, Research, PCSK9, American Heart Association, Gene editing, Eli Lilly and Company, Blood, FDA, AHA, N-Acetylgalactosamine, Cardiovascular disease, Food, Verve, Safety, RNA, Vertex Pharmaceuticals, Therapy, Genetics, Lilly, Patient, LPA, Medicine, Clinical trial, ANGPTL3, Apolipoprotein, Science, LDLR, G&A, IND, Asialoglycoprotein receptor, Moyamoya disease, Human, Beam, Pharmaceutical industry

BOSTON, Feb. 27, 2024 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today reported pipeline updates and financial results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • BOSTON, Feb. 27, 2024 (GLOBE NEWSWIRE) -- Verve Therapeutics , a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today reported pipeline updates and financial results for the fourth quarter and year ended December 31, 2023.
  • “2023 was a momentous year for Verve as we made significant strides towards our mission of protecting the world from cardiovascular disease.
  • Verve expects to initiate the VERVE-201 Phase 1b clinical trial in the second half of 2024, subject to regulatory clearances.
  • In October 2023 , Verve announced the expansion of its relationship with Lilly, which was previously established in June 2023.

Vaxxinity’s Cholesterol Vaccine Candidate Successfully Lowers LDL-C: Preclinical Data Published

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Jeudi, février 15, 2024
PCSK9, Toxicity, Pathology, Moyamoya disease, CAPE, Stroke, Life, Antibody, Risk, Safety, Lipid, Journal, Apolipoprotein, Heart, Atherosclerosis, LDLR, Hypercholesterolemia, Solid lipid nanoparticle, Immune system, Pharmaceutical industry

CAPE CANAVERAL, Fla., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Vaxxinity, Inc. (Nasdaq: VAXX), a U.S. company pioneering the development of a new class of medicines, today announced the publication of data from multiple non-human primate studies demonstrating that VXX-401 reproducibly lowers low-density lipoprotein cholesterol (LDL-C) in non-human primates. The results, which support the continued clinical development of VXX-401 as a candidate for the treatment of hypercholesterolemia and prevention of atherosclerotic cardiovascular disease, were published in the Journal of Lipid Research (Volume 65, Issue 2, 100497, February 2024).

Key Points: 
  • Preclinical data published in the Journal of Lipid Research indicate VXX-401 is well-tolerated, with no safety signals observed, and robustly reduces LDL-C in cynomolgus monkeys.
  • The results, which support the continued clinical development of VXX-401 as a candidate for the treatment of hypercholesterolemia and prevention of atherosclerotic cardiovascular disease, were published in the Journal of Lipid Research (Volume 65, Issue 2, 100497, February 2024).
  • “Vaxxinity is committed to providing scalable, accessible, game-changing solution for worldwide heart health,” said Mei Mei Hu, CEO of Vaxxinity.
  • A cholesterol vaccine like VXX-401 may provide a cost-effective and widely deployable solution that could potentially benefit hundreds of millions of people at risk.

1 in 25 carries a genotype that is associated with a shortened lifespan

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Mercredi, novembre 15, 2023
Icelanders, ACMG, Disclosure, Genetics, Breast, Gene, Genetic distance, Proteomics, Risk, Disease, Cholesterol, Cardiovascular disease, Paper, DSM-IV codes, Icelandic, Medicine, Prostate cancer, American College, Genome, Genotype, Survival, Medical genetics, Government, AMGN, Vaccine, Pharmaceutical industry, Genomics, LDLR, Amgen

REYKJAVIK, Iceland, Nov. 15, 2023 /PRNewswire/ -- The study, published today in the New England Journal of Medicine, focuses on genotypes that increase the risk of a disease for which preventive or therapeutic measures have been established. These genotypes are termed actionable genotypes. The scientists used a population-based data set, consisting of 58,000 whole-genome sequenced Icelanders, to assess the fraction of individuals carrying actionable genotypes.

Key Points: 
  • The scientists used a population-based data set, consisting of 58,000 whole-genome sequenced Icelanders, to assess the fraction of individuals carrying actionable genotypes.
  • The study assessed the relationship between actionable genotypes and the lifespan of their carriers.
  • A pathogenic variant in BRCA2, predisposing to breast, ovarian and pancreatic cancer, shortened lifespan by seven years and a variant in LDLR, which causes high levels of cholesterol and cardiovascular disease, shortened lifespan by six years.
  • The researchers determined that 1 in 25 individuals carried an actionable genotype and have, on average, a shortened lifespan.

1 in 25 carries a genotype that is associated with a shortened lifespan

Retrieved on: 
Mercredi, novembre 15, 2023
Icelanders, ACMG, Disclosure, Genetics, Breast, Gene, Genetic distance, Proteomics, Risk, Disease, Cholesterol, Cardiovascular disease, Paper, DSM-IV codes, Icelandic, Medicine, Prostate cancer, American College, Genome, Genotype, Survival, Medical genetics, Government, AMGN, Vaccine, Pharmaceutical industry, Genomics, LDLR, Amgen

REYKJAVIK, Iceland, Nov. 8, 2023 /PRNewswire/ -- The study, published today in the New England Journal of Medicine, focuses on genotypes that increase the risk of a disease for which preventive or therapeutic measures have been established. These genotypes are termed actionable genotypes. The scientists used a population-based data set, consisting of 58,000 whole-genome sequenced Icelanders, to assess the fraction of individuals carrying actionable genotypes.

Key Points: 
  • The scientists used a population-based data set, consisting of 58,000 whole-genome sequenced Icelanders, to assess the fraction of individuals carrying actionable genotypes.
  • The study assessed the relationship between actionable genotypes and the lifespan of their carriers.
  • A pathogenic variant in BRCA2, predisposing to breast, ovarian and pancreatic cancer, shortened lifespan by seven years and a variant in LDLR, which causes high levels of cholesterol and cardiovascular disease, shortened lifespan by six years.
  • The researchers determined that 1 in 25 individuals carried an actionable genotype and have, on average, a shortened lifespan.

Verve Therapeutics Highlights Recent Company Progress and Reports Second Quarter 2023 Financial Results

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Jeudi, août 10, 2023
Stock, Cardiovascular disease, ANGPTL3, European Society of Cardiology, Medicine, Research, TRL, RNA, Lipoprotein(a), MTV: TRL Christmas, Aortic stenosis, Thrombosis, Congress, Patient, Risk, Blood, LDL, Hart–Scott–Rodino Antitrust Improvements Act, PCSK9, Mouse, United Kingdom, Liver, Eli Lilly and Company, ASCVD, LDLR, ESC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Safety, Stroke, Pharmaceutical industry, Vaccine, Lilly, Verve

BOSTON, Aug. 10, 2023 (GLOBE NEWSWIRE) -- Verve Therapeutics, Inc., a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today reported recent company progress and financial results for the second quarter of 2023.

Key Points: 
  • Lp(a) Program Advancing in Collaboration with Lilly; Received $60 Million in Combined Upfront Payment and Equity Investment in August 2023
    BOSTON, Aug. 10, 2023 (GLOBE NEWSWIRE) -- Verve Therapeutics, Inc., a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today reported recent company progress and financial results for the second quarter of 2023.
  • “We are dedicated to bringing life-changing, once-and-done medicines to patients with cardiovascular disease,” said Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve.
  • Finally, we are thrilled to advance our Lp(a) program in collaboration with Lilly, an industry leader in cardiometabolic disease.
  • Based on the progress of the heart-1 clinical trial, the company expects enrollment to be completed outside the United States.