ANGPTL3

CRISPR Therapeutics Provides Business Update and Reports First Quarter 2024 Financial Results

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Wednesday, May 8, 2024
Sickle cell disease, T1D, SHR, Cell, CRISPR, Partnership, CD19, CRISPR Therapeutics, Biomarker, Autoimmunity, SCD, LNP, Lupus, Kingdom, Porphobilinogen, ADC, Eye, ANGPTL3, RAAS, PBG, Therapy, Lymphoid leukemia, Vertex Pharmaceuticals, Type 1 diabetes, Pain, Cas9, AHP, Diabetes, Research, ALA, BP, CD70, AGT, Clinical trial, Government, G&A, TDT, Sale, CRSP, ALAS1, European, Disease, Patient, Liver, Gene, Society, Stem cell, Cardiovascular disease, SLE, Pharmaceutical industry

CASGEVY is the first therapy to emerge from a strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals established in 2015.

Key Points: 
  • CASGEVY is the first therapy to emerge from a strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals established in 2015.
  • CRISPR Therapeutics has two next-generation approaches with the potential to significantly expand the addressable population with SCD and TDT.
  • G&A Expenses: General and administrative expenses were $18.0 million for the first quarter of 2024, compared to $22.4 million for the first quarter of 2023.
  • Collaboration Expense: Collaboration expense, net, was $47.0 million for the first quarter of 2024, compared to $42.2 million for the first quarter of 2023.

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

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Wednesday, May 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.

ProQR Announces Year End 2023 Operating and Financial Results

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Wednesday, March 13, 2024
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(Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2023, and provided a business update.

Key Points: 
  • (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2023, and provided a business update.
  • At December 31, 2023, ProQR held cash and cash equivalents of €118.9 million, compared to €94.8 million at December 31, 2022.
  • The Company experienced a net positive cash flow from operating activities in 2023 primarily due to the receipt of the Lilly up-front payment of $60 million in February 2023.
  • For further financial information for the period ended December 31, 2023, please refer to our 2023 Annual Report on Form 20-F and our Statutory Annual Report which will be available on our website, www.

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

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Tuesday, February 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.

CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, February 21, 2024
Sale, CD19, Lupus, RNA, EAP, LNP, Liver, Cardiovascular disease, CRISPR Therapeutics, CRSP, Therapy, Vertex Pharmaceuticals, Sickle cell disease, Autoimmunity, T1D, G&A, Bill, Partnership, Pharmacology, Guide, SCD, TDT, Pharmacokinetics, Clinical trial, ANGPTL3, United, Hypertriglyceridemia, Medicaid, Research, Human, CRISPR, Gene editing, CD70, Stem cell, ADC, Risk, Genetics, Patient, SLE, KSA, Moyamoya disease, Pharmaceutical industry

R&D Expenses: R&D expenses were $95.1 million for the fourth quarter of 2023, compared to $103.6 million for the fourth quarter of 2022.

Key Points: 
  • R&D Expenses: R&D expenses were $95.1 million for the fourth quarter of 2023, compared to $103.6 million for the fourth quarter of 2022.
  • G&A Expenses: General and administrative expenses were $16.5 million for the fourth quarter of 2023, compared to $21.2 million for the fourth quarter of 2022.
  • Collaboration Expense: Collaboration expense, net, was $20.0 million for the fourth quarter of 2023, compared to $6.8 million for the fourth quarter of 2022.
  • Net Income (Loss): Net income was $89.3 million for the fourth quarter of 2023, compared to a net loss of $110.6 million for the fourth quarter of 2022.

ProQR Highlights New Platform Data from Presentation on Axiomer™ RNA Editing Technology at Deaminet 2024

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Friday, January 19, 2024
EON, RNA-Seq, Oligonucleotide, Knowledge, RNA, Science, Circulatory system, Mouse, NHP, NTCP, ACTB, Cholestasis, ANGPTL3, Cardiovascular disease, Plasma, Conference, B4GALT1, LDL, Liver, Eon, ProQR, ADAR, Nested polymerase chain reaction, CNS, Video game

“We were pleased to present new in vivo data for our proprietary Axiomer RNA editing technology platform at the Deaminet 2024 meeting, demonstrating robust editing of ACTB in the liver of NHP, as well as functional protein data with the liver target ANGPTL3 in mice,” said Gerard Platenburg, Chief Scientific Officer of ProQR.

Key Points: 
  • “We were pleased to present new in vivo data for our proprietary Axiomer RNA editing technology platform at the Deaminet 2024 meeting, demonstrating robust editing of ACTB in the liver of NHP, as well as functional protein data with the liver target ANGPTL3 in mice,” said Gerard Platenburg, Chief Scientific Officer of ProQR.
  • “To date, we have generated robust in vitro and in vivo preclinical platform data across a range of liver and CNS targets.
  • Presentation of new data highlighting up to 70% editing efficiency of ACTB in liver of mice and NHPs at multiple timepoints.
  • These new preclinical platform data further highlight the potential of Axiomer EONs in preparation for clinical development and supporting discovery of potential new therapeutic applications.

CRISPR Therapeutics Highlights Strategic Priorities and 2024 Outlook

Retrieved on: 
Monday, January 8, 2024
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ZUG, Switzerland and BOSTON, Jan. 08, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today highlighted its strategic priorities and 2024 outlook as the Company enters its next phase of growth.

Key Points: 
  • ZUG, Switzerland and BOSTON, Jan. 08, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today highlighted its strategic priorities and 2024 outlook as the Company enters its next phase of growth.
  • Exa-cel is the first therapy to emerge from a strategic partnership between CRISPR Therapeutics and Vertex Pharmaceuticals.
  • Vertex leads global development, manufacturing, regulatory and commercialization of CASGEVY with support from CRISPR Therapeutics.
  • CRISPR Therapeutics continues to focus on resource efficiency and return on invested capital as it advances multiple clinical programs across its pipeline.

Ultragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza® ▼ (evinacumab) for Adolescents and Adults Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)

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Thursday, January 4, 2024
Marketing, Cholesterol, Ultragenyx, NHS England, Adolescence, Hammersmith Hospital, Diet, Ageing, Clinical trial, Trauma, Heart UK, Patient, NHS, ANGPTL3, Lipid, Pharmaceutical industry

Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH).

Key Points: 
  • Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH).
  • Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for this rare and debilitating condition.
  • "We are very pleased that NICE has approved the use of evinacumab within the NHS.
  • The NICE committee acknowledged that clinical trial evidence shows that Evkeeza, combined with other lipid-lowering therapies (LLTs), can lower LDL-C levels when statins and other LLTs have not reduced them enough.

Human medicines European public assessment report (EPAR): Evkeeza, evinacumab, Date of authorisation: 17/06/2021, Revision: 3, Status: Authorised

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Tuesday, January 2, 2024
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Human medicines European public assessment report (EPAR): Evkeeza, evinacumab, Date of authorisation: 17/06/2021, Revision: 3, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Evkeeza, evinacumab, Date of authorisation: 17/06/2021, Revision: 3, Status: Authorised

Ultragenyx Receives European Commission Decision for Evkeeza® (evinacumab) Expanded Indication in Children Aged 5 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)

Retrieved on: 
Monday, December 18, 2023
European Commission, Disorder, Apolipoprotein, Diet, Ageing, Civil service commission, Committee, Patient, ANGPTL3, Pharmaceutical industry

Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for children as young as 5 years old to control dangerously high levels of low-density lipoprotein cholesterol (LDL-C) caused by HoFH.

Key Points: 
  • Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for children as young as 5 years old to control dangerously high levels of low-density lipoprotein cholesterol (LDL-C) caused by HoFH.
  • Evkeeza initially received EC decision as an adjunct to diet and other lipid-lowering therapies in adolescents and adults aged 12 years and older with HoFH in June 2021.
  • With the addition of Evkeeza, children (n=14) were able to reduce their LDL-C by 48% at week 24 on average.
  • The treatment is now reimbursed and commercially available to prescribe for appropriate patients with HoFH in the U.S., Canada, Italy and Germany.