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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. In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs).
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.
Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). Our pipeline of programmes focused on neuromuscular and cardiac rare disease gives us deep insight into the diverse and complex nature of dilated cardiomyopathy.”.
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. Life Edit will get an upfront payment and is eligible for potential milestone payments.
It is difficult to control where the viral vectors insert genes in the genome, and it is difficult to manufacture large quantities of clinical-grade viral vectors. The goal of the team was to significantly increase yields of correctly engineered immune cells with CRISPR to effectively manufacture cell therapies.
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. As per our analysis, majority of the kits use purified RNA / DNA as the input sample. Nanoparticles Contract Manufacturing.
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.
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.
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. Formulas can be generated in days and clinical-grade manufacturing can be accomplished in weeks. RNA technology is not new nor has its potential been surprising.
PH1 is an ultra-rare geneticdisease characterized by oxalate overproduction. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
The company’s commitment to SMA extends beyond gene therapy to branaplam (LMI070), an oral, once-weekly RNA splicing modulator also currently under development, to expand the treatment options for SMA patients. . More than 30% of patients with SMA Type 2 will die by age 25.
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. In the third quarter of 2023, the company’s revenue surged to $63.7
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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