Porphobilinogen

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

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수요일, 5월 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.

CRISPR Therapeutics Highlights ASGCT Oral Presentation and Announces New Programs Utilizing In Vivo Gene Editing Approach

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수요일, 5월 8, 2024
Pain, CRSP, Patient, SHR, Cas9, Cell, CRISPR, ALAS1, LNP, AHP, Gene editing, Angiotensin, Paratriathlon, RNA, IOP, ALA, Disease, Porphobilinogen, Liver, CRISPR Therapeutics, Gene, Eye, Society, Trabecular meshwork, MYOC, RAAS, Cardiovascular disease, BP, AGT, Biomarker, PBG, Therapy, Glaucoma

In addition, CRISPR Therapeutics announced the expansion of its in vivo pipeline with two new programs.

Key Points: 
  • In addition, CRISPR Therapeutics announced the expansion of its in vivo pipeline with two new programs.
  • “The expansion of our in vivo pipeline speaks to the scalability of the platform and the exceptional translation capabilities of our team.
  • The addition of two more programs, CTX340 and CTX450, utilizing this LNP delivery technology demonstrates the modularity and scalability of the platform.
  • In today’s presentation, the Company presented data demonstrating efficient and specific delivery to TM cells in mouse, non-human primate, and ex vivo human eyes.