RNA transfection

Phase 1 Clinical Data of Milademetan Published in Journal of Clinical Oncology

Retrieved on: 
Monday, January 23, 2023
Bachelor of Medicine, Bachelor of Surgery, MANTRA, Journal, MD, Patient, DD, MDM4, CDK4, Journal of Clinical Oncology, Cytopenia, Biomarker, Therapy, Neoplasm, RAIN, Progression-free survival, RNA transfection, Doctor of Philosophy, MDM2, Pharmaceutical industry

NEWARK, Calif., Jan. 23, 2023 (GLOBE NEWSWIRE) -- Rain Oncology Inc. (NasdaqGS: RAIN), (“Rain”), a late-stage company developing precision oncology therapeutics with its lead product candidate, milademetan, an oral, small molecule inhibitor of the p53-MDM2 complex that reactivates p53, today announced the publication of a peer-reviewed article titled, “A First-in-Human Phase I Study of Milademetan, an MDM2 Inhibitor, in Patients with Advanced Liposarcoma, Solid Tumors or Lymphomas” in the Journal of Clinical Oncology. Phase 1 clinical data in the paper highlight the activity and tolerability using intermittent dosing of milademetan across a range of tumor types including dedifferentiated liposarcoma (DD LPS), which represented the largest proportion of patients enrolled in the study (n=53).

Key Points: 
  • Phase 1 clinical data in the paper highlight the activity and tolerability using intermittent dosing of milademetan across a range of tumor types including dedifferentiated liposarcoma (DD LPS), which represented the largest proportion of patients enrolled in the study (n=53).
  • “An intermittent dosing schedule (260 mg qd, 3/14 days) of our highly selective inhibitor of the p53-MDM2 complex, milademetan, resulted in favorable safety and clinical activity in the Phase 1 trial in DD LPS patients,” said Robert Doebele, MD, Ph.D., co-founder, president and chief scientific officer of Rain.
  • The preferred intermittent dosing schedule of milademetan (260 mg qd 3/14 days) mitigates dose-limiting hematologic adverse events while maintaining activity, leading to:
    Fewer dose reductions (21.1%; n=8) and dose interruptions (15.8%; n=6) compared with extended/continuous schedules (23.3%; n=16 and 34.8%; n=24, respectively).
  • Preliminary single-agent activity with milademetan in DD LPS prompted the ongoing, randomized Phase 3 MANTRA trial (NCT04979442), with topline data anticipated in the first quarter of 2023.

aTyr Pharma Gains E.U. Patent Covering Use of Efzofitimod with Pirfenidone for Lung Inflammation or Fibrosis

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Thursday, January 19, 2023
ATyr Pharma, RNA transfection, Pirfenidone, European Patent Office, European Patent Convention, Lung, Biology, Interstitial lung disease, Idiopathic pulmonary fibrosis, Therapy, Patent, ILD, Research, Sarcoidosis, Fibrosis, SAN, Inflammation, EPO, HARS, IPF, N, Pharmaceutical industry

18 787408.6, titled, “Compositions and methods for treating lung inflammation,” covers the use of the company’s lead therapeutic candidate, efzofitimod, in combination with the anti-fibrotic agent pirfenidone.

Key Points: 
  • 18 787408.6, titled, “Compositions and methods for treating lung inflammation,” covers the use of the company’s lead therapeutic candidate, efzofitimod, in combination with the anti-fibrotic agent pirfenidone.
  • A patent from the recently allowed application is expected to be issued in the coming months.
  • aTyr is a biotherapeutics company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform.
  • aTyr’s primary focus is efzofitimod, a clinical-stage product candidate which binds to the neuropilin-2 receptor and is designed to downregulate immune engagement in fibrotic lung disease.

European Commission Grants Orphan Drug Designation for aTyr Pharma’s Efzofitimod for Treatment of Sarcoidosis

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Wednesday, January 18, 2023
Disease, ATyr Pharma, RNA transfection, NRP2, Inflammation, Prognosis, Union, European Medicines Agency, Royal commission, Death, Quality of life, Committee, Safety, Sarcoidosis, Biomarker, FDA, Scar, Granuloma, Biology, Marketing, COMP, Diagnosis, Patient, Prevalence, Degenerative disease, Research, Neuropilin, Fibrosis, SAN, Medicine, FC, EMA, European Commission, EC, Pharmaceutical industry, EU

“The EC’s decision to grant orphan drug designation to efzofitimod acknowledges the unmet medical need in sarcoidosis for the approximately 150,000 people in the European Union (EU) living with this chronic, debilitating disease,” said Sanjay S. Shukla, M.D., M.S., President and CEO of aTyr.

Key Points: 
  • “The EC’s decision to grant orphan drug designation to efzofitimod acknowledges the unmet medical need in sarcoidosis for the approximately 150,000 people in the European Union (EU) living with this chronic, debilitating disease,” said Sanjay S. Shukla, M.D., M.S., President and CEO of aTyr.
  • “The criteria for orphan status in the EU go beyond the rarity of the disease.
  • Efzofitimod received orphan drug and Fast Track designations for sarcoidosis from the United States Food and Drug Administration (FDA) in 2022.
  • aTyr is currently conducting EFZO-FIT™, a Phase 3 study of efzofitimod in pulmonary sarcoidosis patients.

Piezo Therapeutics Launches to Transform Delivery of Nucleic Acid Medicines

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Thursday, January 19, 2023
Gene expression, VentureLab, COVID, Global health, Therapy, COVID-19, RNA transfection, Open philanthropy (doctrine), DNA, Safety, Science Publishing Group, Vaccine, Drug development, Partnership, Engineering, Georgia Tech Research Institute, Regent, RNA, National Academy of Inventors, Sundell, National academy, Medical device, Management, Pharmaceutical industry, Piezo, Georgia Tech

ATLANTA, Jan. 19, 2023 /PRNewswire-PRWeb/ -- Piezo Therapeutics launched with $2 million in seed funding from Open Philanthropy to develop its platform: a simple and affordable way of delivering RNA and DNA medicines, including vaccines and therapeutic proteins. Piezo's platform combines electric pulses and microneedles to deliver nucleic acid medicines safely and efficiently while accelerating manufacturing, easing access, and minimizing costs.

Key Points: 
  • Piezo is developing a simple, affordable, and scalable platform designed for rapid, safe, and efficient delivery of nucleic acid medicines, including RNA/DNA vaccines, therapeutic proteins, and dermal gene therapies.
  • Invented by Georgia Tech researchers, Piezo launches with $2 million in seed funding from Open Philanthropy.
  • Piezo's platform combines electric pulses and microneedles to deliver nucleic acid medicines safely and efficiently while accelerating manufacturing, easing access, and minimizing costs.
  • While this platform has high applicability for vaccines (Piezo's immediate focus), it is designed for a broad array of nucleic acid therapeutics.

Kaneka expands GMP manufacturing capacity for mRNA in Kaneka Eurogentec S.A.

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Thursday, January 19, 2023
Biotechnology, Pharmaceutical, Other Manufacturing, Health, COVID-19, Infectious Diseases, Genetics, Manufacturing, Food and Drug Administration, Kaneka Corporation, Nucleotide, RNA transfection, Organic acid, DNA, Corporation, Animal, FDA, Bacteria, Vaccine, Infection, Protein, Cell division, Growth, Therapy, Escherichia coli, A-DNA, RNA, JPY, Research, Cancer, Good manufacturing practice, CDMO, Cosmetics, Health care, Peptide, Multimedia, Good, GMP, Fine chemical

Kaneka Eurogentec S.A. will grow as a leading company of biopharmaceutical CDMO*3, responding to the vigorous global demand for mRNA.

Key Points: 
  • Kaneka Eurogentec S.A. will grow as a leading company of biopharmaceutical CDMO*3, responding to the vigorous global demand for mRNA.
  • The demand for GMP manufacturing of mRNA will increase drastically with the active development of biopharmaceutical pipelines by pharmaceutical companies worldwide.
  • Kaneka Eurogentec S.A. is a CDMO for biopharmaceuticals inspected by the US FDA*4 and has world-class technology for producing plasmid DNA*5 and GMP manufacturing track records of over 25 years.
  • In addition, Kaneka Eurogentec S.A. has started GMP manufacturing services for mRNA since 2020, and this capacity expansion will be utilized to expand the CDMO business.

Coming in From the Cold: Needle-free Patch Technology for Mrna Vaccines Aims to End Need for Frozen Storage and Improve Access

Retrieved on: 
Tuesday, January 17, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, COVID-19, RNA transfection, Heart, Public health, Safety, LMIC, Coalition for Epidemic Preparedness Innovations, Vaccination, Skin, Vaccine, Partnership, Infection, Developing country, Epidemic, Coalition, Policy, Investment, School, Pharmaceutical industry, CEPI

When applied to the skin, the patch delivers vaccine to the abundant immune cells immediately below the skin surface.

Key Points: 
  • When applied to the skin, the patch delivers vaccine to the abundant immune cells immediately below the skin surface.
  • CEPI and Vaxxas are committed to enabling global equitable access to the vaccines they develop including those incorporating patch technology.
  • One possible way to improve access to mRNA vaccines in future would be to adapt their formulation so that they could be delivered using microarray patches.
  • This removes the need for frozen storage, allows for easier distribution and safe delivery with accurate dosing.

Global mRNA Synthesis and Manufacturing Services Markets, 2022-2035: Market Landscape and Future Opportunity for the Players Involved in the Synthesis and Manufacturing of Research- and GMP-grade mRNA - ResearchAndMarkets.com

Retrieved on: 
Monday, January 16, 2023
Biotechnology, Genetics, Health, RNA transfection, Biosynthesis, Toxicity, Administration, COVID-19, BioNTech, FDF National Camp, Thermo Fisher Scientific, Type, Degenerative disease, Vaccine, API, Infection, Growth, Therapy, CureVac, RNA, GMP, Fine chemical, Pharmaceutical industry, New England Biolabs, Eurogentec

The "mRNA Synthesis and Manufacturing Services Market (2nd Edition), 2022-2035" report features an extensive study of the current market landscape and future opportunity for the players involved in the synthesis and manufacturing of research- and GMP-grade mRNA for use in the development of therapeutics and vaccines.

Key Points: 
  • The "mRNA Synthesis and Manufacturing Services Market (2nd Edition), 2022-2035" report features an extensive study of the current market landscape and future opportunity for the players involved in the synthesis and manufacturing of research- and GMP-grade mRNA for use in the development of therapeutics and vaccines.
  • One of the key objectives of the report was to estimate the current opportunity and the future growth potential of the mRNA synthesis and contract manufacturing services market over the coming years.
  • Presently, more than 195 mRNA therapeutics / mRNA vaccines are under development or commercialized for the treatment of a variety of indications.
  • Specifically, in terms of type of product, the market is anticipated to be driven by contract manufacturing of mRNA vaccines.

Outlook on the RNA Analysis Global Market to 2028 - Featuring Illumina, Bio-Rad Laboratories, Eurofins Scientific and Merck Among Others - ResearchAndMarkets.com

Retrieved on: 
Wednesday, January 11, 2023
Biotechnology, Genetics, Health, Classification, Genome, Gene expression, RNA, Phenotype, Toxicity, RNA transfection, DNA, Personalized medicine, Molecular biology, Orthornavirae, Microarray, Biomarker, Collection, Transcriptome, Research, Vaccine, Gene, Drug development, Protein, RNA-Seq, Application, MicroRNA, Drug discovery, Cancer, CVD, Data analysis, Real-time, Real, Toxicology, Healthcare

The Global RNA Analysis Market size is expected to reach $28 billion by 2028, rising at a market growth of 15.6% CAGR during the forecast period.

Key Points: 
  • The Global RNA Analysis Market size is expected to reach $28 billion by 2028, rising at a market growth of 15.6% CAGR during the forecast period.
  • The method used to produce RNA in a lab setting from a DNA template is known as in vitro transcription.
  • For use in procedures like in vitro translation, the investigation of RNA viruses, and RNA: protein interaction investigations, RNA is produced from DNA templates using in vitro transcription devices.
  • For use in RNA analysis research, single-stranded RNA probes, microgram amounts of specified RNA transcripts, and antisense RNA are all prepared using riboprobe systems.

ReCode Therapeutics Announces Strategic Investment from the Cystic Fibrosis Foundation to Accelerate Development of Novel mRNA Therapy

Retrieved on: 
Tuesday, January 10, 2023
Science, Other Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, IND, Valencia CF Foundation, Therapy, Cystic fibrosis, RNA transfection, Recode, CFTR, Cystic Fibrosis Foundation, SORT, LNP, Conference, Patient, Investigational New Drug, CF, MBA, Vaccine, Pharmaceutical industry

“We are excited to expand our relationship with the CF Foundation with this investment in our CFTR mRNA program for cystic fibrosis,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics.

Key Points: 
  • “We are excited to expand our relationship with the CF Foundation with this investment in our CFTR mRNA program for cystic fibrosis,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics.
  • The investment includes a commitment by the CF Foundation to invest another $5 million upon completion of development milestones.
  • ReCode’s CFTR mRNA therapy aims to compensate for mutated CFTR, the fundamental genetic defect in CF.
  • Preclinical data from ReCode presented at the North American Cystic Fibrosis Conference in 2022 demonstrated that CFTR mRNA encapsulated in SORT LNPs can be nebulized and delivered as an aerosol.

CEPI partners with Tiba Biotech to evaluate next-generation RNA vaccine platform technology to respond to 'Disease X'

Retrieved on: 
Thursday, January 12, 2023
Fever Dream, CEPI, Reactogenicity, Fever, COVID-19, RNA transfection, Yellow fever, G7, LNP, Vaccine, Coalition for Epidemic Preparedness Innovations, Equitable, Partnership, Disease, Refrigeration, RNA, Mortality, Flavivirus, Epidemic, Coalition, US, Venezuelan equine encephalitis virus, Policy, JEV, Control

OSLO Norway and CAMBRIDGE, Mass., Jan. 12, 2023 /PRNewswire/ -- The Coalition for Epidemic Preparedness Innovations (CEPI) and Tiba Biotech , a preclinical biopharmaceutical company, today announced a new partnership agreement to evaluate Tiba Biotech's next-generation RNA vaccine platform technology.

Key Points: 
  • OSLO Norway and CAMBRIDGE, Mass., Jan. 12, 2023 /PRNewswire/ -- The Coalition for Epidemic Preparedness Innovations (CEPI) and Tiba Biotech , a preclinical biopharmaceutical company, today announced a new partnership agreement to evaluate Tiba Biotech's next-generation RNA vaccine platform technology.
  • Tiba Biotech's innovative platform could offer substantial advantages over existing RNA vaccines when responding to future epidemic or pandemic diseases.
  • CEPI will provide US$2 million to advance Tiba Biotech's novel RNA nanoparticle delivery platform, RNABL™.
  • To deliver an RNA payload, Tiba Biotech uses organic molecules to form biodegradable nanoparticles that are less inflammatory than current lipid nanoparticle (LNP) formulations.