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Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.
Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.
Jupiter Neurosciences, a clinical-stage pharma company specializing in neuroinflammation, made its public market debut with an initial public offering (IPO) on the Nasdaq Capital Market under the symbol “JUNS.” ” The company priced 2,750,000 shares of common stock at $4.00
Carlsbad, California-based Ionis Pharmaceuticals announced it will be sharing promising new trial data for its RNA-targeted prophylactic treatment donidalorsen for hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress in San Diego, California.
Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting. Qfitlia is the second RNA interference therapy to receive a highly anticipated FDA approval recently. Qfitlia will be entering a small but competitive market. Sanofi is not new to the hemophilia market.
The collaboration will use the vector biology, manufacturing capabilities and the drug development experience of Solid Biosciences and Phlox Therapeutics’ knowledge in genetic cardiomyopathies and RNA therapies. The post Solid Biosciences partners with Phlox for cardiac disease therapies appeared first on Pharmaceutical Technology.
Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and geneticdiseases.
The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. The market for mRNA-based vaccines and therapeutics is projected to grow from $46.7 RNA technology is not new nor has its potential been surprising.
US biotech, CAMP4 Therapeutics, has secured US$100m in a Series B financing round that it says will be used to accelerate expansion of its regulatory RNA (regRNA) platform.
The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging geneticdiseases.
These kits require less input sample, produce sufficient DNA / RNA libraries with minimum errors and are compatible with a broad range of input samples, including blood, cells, plasma and saliva. The various advantages of NGS are briefly described below: Next Generation Sequencing (NGS) Library Preparation Kits Market – Roots Analysis.
Molecular diagnostic tests refer to tests intended to detect specific sequences in human genomic samples, such as DNA or RNA, in order to diagnose a particular disease. Further, such tests also aim to identify single nucleotide polymorphisms, deletions, rearrangements, and insertions in genetic sequences.
Related: Top 30 Pharma Companies in 2023: Statistics and Trends 1) Moderna Compound annual growth rate: 415 percent Moderna, headquartered in Massachusetts, is a prominent biotechnology company specializing in RNA therapeutics, particularly mRNA vaccines. vaccine, mRNA-1273.815.
The fully integrated pharmaceutical company creates value through China’s specialty pharmaceutical markets with focus on iron deficiency, pain management and respiratory. Cornering the untapped Chinese CNS market, SciNeuro launched with $100 million in their pocket. SciNeuro Pharmaceuticals . Pear Therapeutics.
is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.
PH1 is an ultra-rare geneticdisease marked by overproduction of oxalate, which causes deposits of calcium oxalate crystals in the kidneys and urinary tract and can lead to painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure, and system organ dysfunction. It is an RNA interference (RNAi) therapeutic.
PH1 is an ultra-rare geneticdisease characterized by oxalate overproduction. With the approval of Oxlumo, the FDA has granted Alnylam a pediatric rare disease priority review voucher that entitles the Company to designate a single new drug application to qualify for a priority review in the future.
This does not impact marketed Zolgensma ® (onasemnogene abeparvovec) and the company continues to advance its regulatory filings and intravenous clinical studies. About Novartis Gene Therapies Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare geneticdiseases.
Since then, the field of nanomedicine has steadily progressed to reach high points such as the successful use of nanotechnology to deliver messenger RNA (mRNA)-based Covid-19 vaccines. Langer, now a David H. These could feature in the next four to five years. Free Report How is the Biopharmaceutical industry evolving?
From leveraging artificial intelligence (AI) to streamline diagnostics and treatments to exploring the untapped potential of RNA-based therapeutics, biotechnology is shaping the future of healthcare and beyond. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.
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