CRISPR

Wyss Institute’s AminoX project receives funding from Northpond Labs to accelerate innovation in protein-based therapeutics

Retrieved on: 
Monday, April 8, 2024
Molecule, Research, Innovation, Patient, DNA, Wyss Institute, Engineering, Protein, Laboratory, Organic acid, Harvard University, CRISPR, Drug development, Wyss, Biological engineering, Therapy, Genetic code, FDA, Antiandrogen, Vocabulary, Fine chemical

This is the fourth Wyss project selected by Northpond Labs for additional funding.

Key Points: 
  • This is the fourth Wyss project selected by Northpond Labs for additional funding.
  • The Laboratory has previously funded the Wyss’ eRNA (now being commercialized by EnPlusOne Biosciences ), SomaCode , and Lab-on-a-Molecule projects.
  • “The AminoX platform has the potential to transform our ability to develop differentiated protein-based therapeutics by significantly expanding the vocabulary of building blocks that can be incorporated into biologics at high throughput.
  • We are thrilled that the AminoX team’s journey to the market will be accelerated by this support,” said Angelika Fretzen, Ph.D., M.B.A., the Wyss Institute’s Technology Translation Director & Chief Operating Officer.

Caribou Biosciences Expands Clinical Development of CB-010 with FDA Clearance of IND in Lupus

Retrieved on: 
Thursday, April 4, 2024
Logic, LN, Transplant, Medical College of Wisconsin, Rachel Haurwitz, Infection, Lupus, Risk, Medical college, Patient, Inflammation, Tissue, Autoimmune disease, Standard of care, Doctor of Philosophy, MD, Autoantibody, IND, Congress, HLA, Reindeer, Antibody, CRISPR, Safety, Immune system, Fatigue, FDA, Lupus nephritis, Autoimmunity, Bone marrow, ERL, Medicine, CD19, Food, PD-1, Pharmaceutical industry

BERKELEY, Calif., April 04, 2024 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today announced that it received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) for CB-010, an allogeneic anti-CD19 CAR-T cell therapy with a PD-1 knockout (KO), for the treatment of lupus nephritis (LN) and extrarenal lupus (ERL). The Phase 1, multicenter, open label GALLOP clinical trial of CB-010 in patients with LN and ERL is expected to initiate by year-end 2024.

Key Points: 
  • The Phase 1, multicenter, open label GALLOP clinical trial of CB-010 in patients with LN and ERL is expected to initiate by year-end 2024.
  • CB-010 targets CD19, a protein on the surface of B cells, and has a PD-1 knockout (KO) that reduces CAR-T cell exhaustion.
  • CB-010 holds the potential for deep depletion of disease-causing B cells which could reset the immune system, leading to sustained drug-free remission.
  • Instead, the chRDNA technology allows for precise insertion of the CAR at an intended location within the T cell genome.

TransCode Therapeutics Reports 2023 Results; Provides Business Update

Retrieved on: 
Wednesday, April 3, 2024
Research, Transcoding, Brain, Survival, CFO, TTX, Patient, Operating cost, RNA, Sale, CMC, Cancer, OTS, IND, AACR, Glioblastoma, Pancreatic cancer, Therapy, Genomics, CRISPR, Breast cancer, Melanoma, Debiopharm, FDA, Liver, IRB, Pharmaceutical industry

BOSTON, April 03, 2024 (GLOBE NEWSWIRE) -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today reported financial results for 2023 and recent business progress.

Key Points: 
  • BOSTON, April 03, 2024 (GLOBE NEWSWIRE) -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today reported financial results for 2023 and recent business progress.
  • “We believe 2023 was extremely productive and pivotal for TransCode.
  • The year was highlighted by preliminary clinical results from our Phase 0 clinical trial with radiolabeled TTX-MC138,” said Tom Fitzgerald, interim CEO and CFO of TransCode.
  • We also continued to achieve other important milestones, with the ultimate objective of fulfilling the promise of RNA therapeutics for oncology applications.

CRISPR Therapeutics to Present at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting

Retrieved on: 
Monday, April 1, 2024
Abstract (summary), MD, Cell, Gene, Society, CRSP, Annual general meeting, CRISPR

ZUG, Switzerland and BOSTON, April 01, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting, taking place May 7 – 11, 2024, in Baltimore, MD and virtually.

Key Points: 
  • ZUG, Switzerland and BOSTON, April 01, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting, taking place May 7 – 11, 2024, in Baltimore, MD and virtually.
  • Abstracts will be released to the public on April 22, 2024, at 4:30 p.m.
  • ET on the presentation day, Wednesday May 8, 2024.
  • A copy of the presentation will be available at www.crisprtx.com once the presentation concludes.

Metagenomi Reports Business Updates and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, March 27, 2024
Research, Dicarboxylic acid, AAV, Enzyme, Development, Gene editing, CAST, Cardiovascular disease, Haemophilia, Vivisection, Patient, Primate, RNA, DNA, Wave 1, Sale, Metagenomics, G&A, IPO, Ultra, Doctor of Philosophy, Protein, MBA, Genome, SMART, Factor VIII, CRISPR, Amyloidosis, MGX, AGT, Liver, LNP, Ionis, PAM, Vaccine

EMERYVILLE, Calif., March 27, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived gene editing toolbox, today reported financial results for the full year ended December 31, 2023, and additional business updates.

Key Points: 
  • The aggregate gross proceeds to Metagenomi from the offering were approximately $93.75 million, before deducting underwriting discounts and commissions and offering expenses.
  • In addition, cash used to fund our operations was $91.4 million for the year ended December 31, 2023.
  • R&D Expenses: Research and development (R&D) expenses were $94.4 million for the full year ended December 31, 2023.
  • G&A Expenses: General and administrative (G&A) expenses were $28.8 million for the full year ended December 31, 2023.

Affini-T Therapeutics Presents Preclinical Data from its Oncogenic Driver Programs Targeting KRAS G12D and p53 R175H at the American Association for Cancer Research (AACR) Annual Meeting 2024

Retrieved on: 
Sunday, April 7, 2024
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Toxicity, TP53, Patient, Lung, Arginine, AACR, Cholangiocarcinoma, Therapy, TRAC, Pancreas, CRISPR, TCR, Vaccine

Affini-T Therapeutics, Inc ., a precision immunotherapy company unlocking the power of T cells against oncogenic driver mutations, today announced that management is presenting data from the company’s oncogenic driver programs targeting HLA-A*11:01 KRAS G12D and HLA-A*02:01 p53 R175H at this year’s American Association for Cancer Research (AACR) Annual Meeting 2024 in San Diego.

Key Points: 
  • Affini-T Therapeutics, Inc ., a precision immunotherapy company unlocking the power of T cells against oncogenic driver mutations, today announced that management is presenting data from the company’s oncogenic driver programs targeting HLA-A*11:01 KRAS G12D and HLA-A*02:01 p53 R175H at this year’s American Association for Cancer Research (AACR) Annual Meeting 2024 in San Diego.
  • “We are also proud to present preclinical data from our cell therapy targeting KRAS G12D, which highlights the promising potential of our proprietary non-viral knock-in THRIVE™ platform.
  • The study supports the planned clinical investigation of the novel KRAS G12D mutant TCR-engineered CD4+ and CD8+ T cell therapy in 2024.
  • These data support the planned clinical development of a novel non-viral TRAC-knocked-in T cell therapy for the treatment of p53 R175H-mutant solid tumors.

Intellia Therapeutics Announces First Patient Dosed in the Phase 3 MAGNITUDE Study of NTLA-2001 as a Single-Dose CRISPR-Based Treatment for Transthyretin Amyloidosis with Cardiomyopathy

Retrieved on: 
Monday, March 18, 2024
Pharmacology, Royal Free Hospital, NTLA, Disease, TTR, Richmond Pharmacology, Patient, Amyloidosis, Cardiomyopathy, Intellia Therapeutics, Heart failure, Therapy, CRISPR, Safety, Phase 1, ATTR, Pharmaceutical industry

NTLA-2001 is an investigational in vivo CRISPR-based therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of transthyretin (ATTR) amyloidosis.

Key Points: 
  • NTLA-2001 is an investigational in vivo CRISPR-based therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of transthyretin (ATTR) amyloidosis.
  • The MAGNITUDE trial is evaluating the efficacy and safety of NTLA-2001 in patients with ATTR amyloidosis with cardiomyopathy.
  • We look forward to evaluating the efficacy and safety of NTLA-2001 in patients with cardiomyopathy in the pivotal Phase 3 trial.
  • There is remarkable interest from the ATTR amyloidosis patient community for a potential single-dose treatment, and we look forward to contributing to the advancement of novel treatment approaches.”

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

Retrieved on: 
Thursday, March 14, 2024
Research, Kite Pharma, PIN, CLL, Disease, AID, Patient, EFS, Observation, Pivotal, Cancer, Renal cell carcinoma, Bank statement, News, RCC, Therapy, CRISPR, Projection, MRD, GAAP, Autoimmune disease, Cyclophosphamide, CD19, Pharmaceutical industry

These restated financial statements have no impact on the Company’s cash, cash equivalents and marketable investments, cash runway or business operations.

Key Points: 
  • These restated financial statements have no impact on the Company’s cash, cash equivalents and marketable investments, cash runway or business operations.
  • Research and development expenses were $54.7 million for the fourth quarter of 2023, which includes $7.0 million of non-cash stock-based compensation expense.
  • General and administrative expenses were $17.2 million for the fourth quarter of 2023, which includes $8.2 million of non-cash stock-based compensation expense.
  • Allogene will host a live conference call and webcast today at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time to discuss financial results and provide a business update.

CRISPR Therapeutics Proposes New Appointment to the Board of Directors

Retrieved on: 
Wednesday, March 13, 2024
Development, Gene editing, Growth, Regeneron Pharmaceuticals, Biotechnology, Science and nature, Merck Serono, Patient, Branches of microbiology, Acquisition, Virology, Executive Committee, Max Planck Society, CRISPR Therapeutics, University, Senior, Bayer, Publication, Therapy, Takeda, CRISPR, Amgen, CRSP, Medicine, Pharmaceutical industry

“We are thrilled to welcome Christian to our Board of Directors,” said Samarth Kulkarni, Ph.D., Chief Executive Officer and Chairman of the Board of CRISPR Therapeutics.

Key Points: 
  • “We are thrilled to welcome Christian to our Board of Directors,” said Samarth Kulkarni, Ph.D., Chief Executive Officer and Chairman of the Board of CRISPR Therapeutics.
  • Dr. Rommel currently serves as Executive Vice President, Global Head of Research & Development and a Member of the Executive Committee of Bayer Pharmaceuticals, Inc.
  • He joined Bayer from Roche, where he was most recently Senior Vice President, Global Head of Oncology, Pharma Research and Early Development (pRED).
  • He has authored more than 70 publications, including papers in Science and Nature, and is an inventor or co-inventor of 18 patents.

Allogene Therapeutics and Arbor Biotechnologies Announce Global Gene Editing Licensing Agreement to Support Advancement of Next-Generation Allogeneic CAR T Platform in Autoimmune Disease

Retrieved on: 
Tuesday, March 12, 2024
Development, Gene editing, AID, Patient, SAN, Research, Cancer, Research and development, Imagination, Therapy, CRISPR, Kite Pharma, Autoimmunity, Autoimmune disease, Pharmaceutical industry

“The potential for CAR T as a therapeutic option for autoimmune disease has captured the collective imagination of the scientific community,” said Zachary Roberts, M.D., Ph.D., Executive Vice President of Research & Development and Chief Medical Officer of Allogene.

Key Points: 
  • “The potential for CAR T as a therapeutic option for autoimmune disease has captured the collective imagination of the scientific community,” said Zachary Roberts, M.D., Ph.D., Executive Vice President of Research & Development and Chief Medical Officer of Allogene.
  • “Accordingly, this excitement created a new field for CAR T that has become quickly crowded, making differentiation key for future success.
  • Allogene’s first AID AlloCAR T investigational product is expected to enter Phase 1 clinical trials in early 2025.
  • “We look forward to our collaboration with Allogene as they leverage our extensive gene-editing technologies to develop novel, differentiated allogeneic CAR T therapeutics for autoimmune diseases.”