LNP

Alnylam Highlights Significant Progress with Platform Innovation and Clinical Pipeline at R&D Day

Retrieved on: 
Wednesday, December 13, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, CNS, Hypertension, Cerebral amyloid angiopathy, Hepatocellular carcinoma, Gene, ALN, Cardiomyopathy, RNA interference, IND, Tissue, Liver, Sanofi, Amyloidosis, Gene silencing, Overweight, KHK, Patient, FGF21, CAA, Part, Obesity, T2DM, Neoplasm, Investigational New Drug, Therapy, LNP, Development, Shanda, Growth, HCC, Central nervous system, Type, TTR, Immunotherapy, Polyneuropathy, Haemophilia, Medication, Biology, Coagulopathy, Traffic barrier, Adipose tissue, Vaccine, Medical device, Pharmaceutical industry, Video game, Alnylam Pharmaceuticals, Capella, ATTR

We expect our track record of strong pipeline and commercial execution to continue through the end of 2025 and beyond,” said Akshay Vaishnaw, M.D., Ph.D., Chief Innovation Officer of Alnylam.

Key Points: 
  • We expect our track record of strong pipeline and commercial execution to continue through the end of 2025 and beyond,” said Akshay Vaishnaw, M.D., Ph.D., Chief Innovation Officer of Alnylam.
  • In addition, Alnylam will share an update on delivery advances driving ALN-BCAT, which will soon begin clinical testing in patients with hepatocellular carcinoma (HCC).
  • A peak mean TTR reduction of 97% was achieved at Day 29 and a mean TTR reduction of 93% was sustained at Day 180.
  • The Company’s R&D Day event will be held today, Wednesday, Dec. 13, from 8:30 a.m. to 12:45 p.m.

Almirall and etherna Enter Into a Multi-Target Alliance to Develop mRNA-based Therapies in Medical Dermatology

Retrieved on: 
Tuesday, December 12, 2023
Biotechnology, Other Health, Health, Pharmaceutical, Oncology, Messenger, ALM, Therapy, Skin, LNP, Patient, Vaccine, Almirall

Almirall, S.A. (ALM), a global pharmaceutical company focused on medical dermatology, and etherna, an mRNA/LNP technology platform company, announced today a multi-target alliance to discover and develop new mRNA-based therapies for severe skin diseases, including non-melanoma skin cancer.

Key Points: 
  • Almirall, S.A. (ALM), a global pharmaceutical company focused on medical dermatology, and etherna, an mRNA/LNP technology platform company, announced today a multi-target alliance to discover and develop new mRNA-based therapies for severe skin diseases, including non-melanoma skin cancer.
  • The alliance combines and leverages etherna’s innovative proprietary mRNA capabilities and LNP formulations with Almirall’s leading expertise in medical dermatology to accelerate discovery of novel treatment options.
  • Etherna and Almirall will work collaboratively on the research activities, while Almirall will lead clinical development and commercialization.
  • Under the terms of the agreement, etherna will receive upfront and technology access payments.

BIOVECTRA Launches Specialized Capabilities in mRNA Drug Product Development and Biomanufacturing

Retrieved on: 
Friday, November 17, 2023
Science, Other Science, Biotechnology, Pharmaceutical, Other Manufacturing, Health, Genetics, Manufacturing, Pandemic, Government of Canada, Research, Life, PEI, The Honourable, Government, Atlantic Ocean, Multimedia, Fermentation, Vaccine, Map, Atlantic Canada, NS, Messenger, Therapy, Plasmid, LNP, CGMP, Work, Growth, New Venture Gear, Islander, Education, Pittsburgh Life Sciences Greenhouse, Pharmaceutical industry

View the full release here: https://www.businesswire.com/news/home/20231117977018/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20231117977018/en/
    New Process Development Science Laboratory specializes in mRNA, pDNA, and LNP work (Dartmouth, Nova Scotia, Canada).
  • This suite of BIOVECTRA capabilities specialized in mRNA, pDNA, lipid nanoparticle and fill / finish work is unique and the first of its kind in Canada.
  • From research discovery through to final commercial vials, BIOVECTRA can support clients from biotech start-ups to blue-chip pharmaceutical companies with their mRNA / pDNA drug product projects from its Atlantic Canada-based operations.
  • New Biomanufacturing Centre (Charlottetown, PEI): 36,000 square feet of cGMP manufacturing space for mRNA, pDNA and Lipid Nano Particle formulation.

Innoforce's Partner RNAimmune Granted IND Clearance for RSV mRNA Vaccine from FDA

Retrieved on: 
Monday, December 18, 2023
Safety, GMP, RSV, Food, CDMO, IVT, IND, Patient, FDA, Vaccine, Messenger, Health, Therapy, LNP, Infection, CTD, ATMP

HANGZHOU, China, Dec. 18, 2023 /PRNewswire/ -- RNAimmune, Inc., a partner of Innoforce, announced the U.S. Food and Drug Administration (FDA) granted IND clearance for its RV-1770 Respiratory Syncytial Virus (RSV) mRNA vaccine.

Key Points: 
  • HANGZHOU, China, Dec. 18, 2023 /PRNewswire/ -- RNAimmune, Inc., a partner of Innoforce, announced the U.S. Food and Drug Administration (FDA) granted IND clearance for its RV-1770 Respiratory Syncytial Virus (RSV) mRNA vaccine.
  • As the CDMO partner for RNAimmune, Innoforce provided end-to-end services from plasmid, mRNA IVT, to LNP encapsulation, including process development and optimization, clinical batch GMP manufacturing, and CTD document preparation for the IND submission to the FDA.
  • RNAimmune's RSV mRNA vaccine aims to address this unmet medical need by leveraging mRNA technology.
  • With the FDA's IND clearance, RNAimmune can now commence clinical trials to evaluate the safety and effectiveness of its RSV mRNA vaccine in human subjects.

First In Vivo CAR-M Lead Candidate Nominated Under Carisma-Moderna Collaboration

Retrieved on: 
Thursday, December 14, 2023
Entrepreneurship, Therapy, LNP, IND, Doctor of Pharmacy, Northwest Biotherapeutics, Patient, Myelocyte, Messenger, CARM, SITC, Doctor of Philosophy, Cancer, RNA, Pharmaceutical industry, Moderna, Mitsubishi Carisma

PHILADELPHIA, Dec. 14, 2023 /PRNewswire/ -- Carisma Therapeutics Inc. (Nasdaq: CARM) ("Carisma" or the "Company"), a clinical-stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, today announced the nomination of its first lead candidate under the collaboration with Moderna, Inc. (Nasdaq: MRNA). This first lead candidate will target an antigen present on a solid tumor with significant unmet medical need. This strategic collaboration brings together Carisma's chimeric antigen receptor macrophage (CAR-M) platform with Moderna's messenger RNA (mRNA) and lipid nanoparticle (LNP) technologies to generate and develop in vivo CAR-M therapeutics for oncology.

Key Points: 
  • This first lead candidate will target an antigen present on a solid tumor with significant unmet medical need.
  • This strategic collaboration brings together Carisma's chimeric antigen receptor macrophage (CAR-M) platform with Moderna's messenger RNA (mRNA) and lipid nanoparticle (LNP) technologies to generate and develop in vivo CAR-M therapeutics for oncology.
  • "The delivery of this first candidate demonstrates our ability to create novel in vivo CAR therapies that can be advanced toward the clinic.
  • We look forward to completing IND-enabling studies with the lead candidate and are excited about the prospect of bringing this therapy forward for patients with advanced solid tumors together with Moderna."

Global mRNA Platform Market Research Report 2023-2028: Post COVID-19 Vaccines, mRNA's Promise in Cancer Immunotherapy and Personalized Medicine

Retrieved on: 
Friday, December 1, 2023
Pandemic, Medicine, Attention, Regenerative medicine, Massive parallel sequencing, Pfizer, Immune system, Organization, Haemophilia, Vaccine, RSV, Respiratory syncytial virus, Allergy, Disease, Cancer, Biotechnology, Protein, Patient, DSM-IV codes, Zika virus, Cas9, D-bifunctional protein deficiency, Autoimmune disease, Infection, Messenger, COVID-19, LNP, HIV, BioNTech, CRISPR, Growth, NGS, SARS-CoV-2, Cell, Temperature, Health, RNA

In cancer immunotherapy, for example, mRNA is designed to instruct immune cells to target and attack cancer cells, harnessing the body's immune system to fight the disease.

Key Points: 
  • In cancer immunotherapy, for example, mRNA is designed to instruct immune cells to target and attack cancer cells, harnessing the body's immune system to fight the disease.
  • The Global mRNA Platform Market is poised for remarkable growth, driven by significant advancements, emerging applications, and robust growth drivers.
  • The Global mRNA Platform Market is on an upward trajectory, driven by advancements, emerging applications, and a global response to pandemics.
  • Company Profiles: Detailed analysis of the major companies present in the Global mRNA Platform Market.

ST Pharm Presented '100-Day Expedite Strategy', Preparedness and Proactive Measures for Future Pandemic at Korea-CEPI R&D Workshop

Retrieved on: 
Tuesday, November 21, 2023
CEPI, LMIC, Epidemic, DNA, Technology transfer, CDMO, IVT, IND, FIH, Biotechnology, Vaccine, Infection, Messenger, Therapy, Coalition, LNP, Small RNA, AER, RNA

During the main speaker session, Joo-Sung James Yang, Ph.D., SVP Head of Biotechnology R&D at ST Pharm, addressed the strategy, which aims to develop pandemic vaccines against emerging infectious diseases within 100 days.

Key Points: 
  • During the main speaker session, Joo-Sung James Yang, Ph.D., SVP Head of Biotechnology R&D at ST Pharm, addressed the strategy, which aims to develop pandemic vaccines against emerging infectious diseases within 100 days.
  • ST Pharm highlighted its mRNA platform technologies as a key success factor of '100-Day Expedite Strategy', which are proprietary 5'-capping analogs, marketed as SmartCap®, and LNP drug delivery system, STLNP®.
  • Also, ST PHARM has two different RNA therapeutic biotech companies in the United States, which creates synergistic effects on both CDMO business and new drug/vaccine development.
  • -Vernagen (Atlanta, USA) specialized in the development of mRNA vaccines and antibody-encoding RNA (AER) platform technology for infectious diseases.

Omega Therapeutics Reports Third Quarter 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Thursday, November 9, 2023
EGFR, Research, HCC, NSCLC, AACR, Rheumatism, American Association for the Study of Liver Diseases, OMEGA, IND, International, Therapy, Hepatology, Flagship Pioneering, Liver regeneration, G&A, Investigational New Drug, Conference, Palatine Nos. 1–8, 21, Fibrosis, Dive (Belgian band), MYC, Hepatitis, R, Pharmacokinetics, Acute respiratory distress syndrome, Omega, Epigenomics, Lung, Tissue, Annual report, Messenger, LNP, Institutional review board, Inflammation, Lung cancer, Hepatocellular carcinoma, Cancer, Patient, Gene expression, AASLD, Management, Pharmaceutical industry, Vaccine, Dietary supplement

As of September 30, 2023, the Company had cash, cash equivalents and marketable securities totaling $89.3 million.

Key Points: 
  • As of September 30, 2023, the Company had cash, cash equivalents and marketable securities totaling $89.3 million.
  • Research and development (R&D) expenses for the third quarter of 2023 were $16.5 million, compared to $20.7 million for the third quarter of 2022.
  • General and administrative (G&A) expenses for the third quarter of 2023 were $7.9 million, compared to $5.2 million for the third quarter of 2022.
  • Net loss for the third quarter of 2023 was $22.2 million, compared to $25.8 million for the third quarter of 2022, driven predominantly by a decrease in R&D expenses.

Gritstone bio Reports Third Quarter 2023 Financial Results and Provides Corporate Updates

Retrieved on: 
Wednesday, November 8, 2023
PCV, SLATE, CORAL, FDA, Biomedical Advanced Research and Development Authority, Research, IND, Gritstone bio, NCI, Food, Health, Infection, Vaccine, Human services, National Institutes of Health Clinical Center, Administration for Strategic Preparedness and Response, HIV, Gates, BARDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, United States Department of Health and Human Services, Administration, Development, Gilead, NAB, Clinical trial, COVID-19, National Cancer Institute, Messenger, Collaboration, Immunotherapy, Safety, Office of Inspector General, U.S. Department of Health and Human Services, Cancer, CEPI, Chemistry, Patient, Pharmaceutical industry, Gritstone, LNP

EMERYVILLE, Calif., Nov. 08, 2023 (GLOBE NEWSWIRE) -- Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company working to develop the world’s most potent vaccines, today reported financial results for the third quarter ended September 30, 2023 and provided recent corporate and clinical updates.

Key Points: 
  • In oncology, preliminary findings from the randomized Phase 2/3 study of our personalized cancer vaccine, GRANITE, in MSS-CRC are rapidly approaching.
  • The scientific bedrock of GRANITE extends to SLATE, where our collaboration with Steven A. Rosenberg and the NCI is now advancing.
  • Gritstone met its enrollment target of 100 patients randomized in August 2023.
  • An IND to run a Phase 1 study was cleared by the U.S. Food and Drug Administration (FDA) in October 2023.

Beam Therapeutics Reports Pipeline and Business Updates and Third Quarter 2023 Financial Results

Retrieved on: 
Wednesday, November 8, 2023
Stem cell, FDA, Eli Lilly and Company, IND, Food, SCD, Trial of the century, Hematology, Biotechnology, LNP Media Group, Congress, Business development, G&A, Health, Thalassemia, Liver, Research and development, Sickle cell disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Research, CD34, Piz, R, Nature, Growth, Cell, Clinical trial, Gene, Partnership, LNP, PCSK9, ESGCT, AAT, Transplant, General Administration of Sport of China, AATD, Stock, GMP, Safety, European Society for Primary Immunodeficiencies, Investment, ANGPTL3, Patient, Drug discovery, Toxicity, Potential future exposure, ESCAPE, Administration, Cardiovascular disease, Pharmaceutical industry, Medical imaging, Vaccine, Lilly, Beam, Verve

CAMBRIDGE, Mass., Nov. 08, 2023 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported third quarter 2023 financial results and provided an update on the business and its clinical and pipeline progress.

Key Points: 
  • Beam anticipates that currently consented patients are sufficient to both fill the sentinel cohort (n=3) and to initiate the expansion cohort.
  • Research & Development (R&D) Expenses: R&D expenses were $100.0 million for the third quarter of 2023, compared to $85.3 million for the third quarter of 2022.
  • General & Administrative (G&A) Expenses: G&A expenses were $25.4 million for the third quarter of 2023, compared to $21.8 million for the third quarter of 2022.
  • Net Loss: Net loss was $96.1 million for the third quarter of 2023, or $1.22 per share, compared to $109.6 million for the third quarter of 2022, or $1.56 per share.