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ReviR Therapeutics has signed a research collaboration and option-to-license agreement with Asieris Pharmaceuticals to discover new oncology therapeutics. The company’s VoyageR AI platform integrates computational methods to drug traditionally undruggable RNA targets.
With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.
These efforts aim to fast-track commercialization and out-licensing agreements in one of the world’s most promising pharmaceutical markets. CAMP4’s RNA-based therapies focus on genetic diseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder.
MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The option licensing agreement is based on early-stage clinical results.
Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The system comprises the Cas9 enzyme and a guide RNA. The gene-editing technology allows for precise, directed changes to genomic DNA.
In 2016, it was licensed by the company to Amgen. The siRNA therapy is created to reduce lipoprotein(a) [Lp(a)] levels, which is a genetically decided, independent cardiovascular disease risk factor. The rights to development, regulatory and sales milestone payments of $400m due from Amgen will be retained by Arrowhead.
Biogen has made a further push into RNA-based drug discovery via a collaboration with Envisagenics, which applies artificial intelligence RNA sequencing data to discover new drug targets and therapeutics. At least 370 genetic disorders are known to derive from errors introduced during this splicing process.
Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.
Innova Medical Group, the world’s largest COVID-19 test provider, has secured the licensing rights for commercializing a new COVID-19 testing technology developed at the University of Birmingham. Innova Medical Group, which secured the licensing rights for the test, is the world’s largest provider of lateral flow tests.
Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018.
According to the US biopharma company, genetic analyses of publicly available sequences for the new variants of SARS-CoV-2 suggest the mutations in the viral spike protein that have made them more transmissible shouldn’t affect the way Veklury (remdesivir) works. “We
In November, it signed a licensing deal with Chinese tech giant Baidu to partner on its artificial intelligence platform, which has been developed specifically to optimise mRNA sequences for mRNA-based therapeutics. Dormitzer comes from Pfizer, where he was chief scientific officer for RNA and viral vaccines for more than six years.
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.
Their technology is based on synthetic messenger RNA – short for ribose nucleic acid – which is a short transcript of a longer DNA code. As it’s just a messenger molecule, it does not affect the body’s own genetic code when it is injected as a vaccine – but what it does do is instruct cells to code for copies of a certain protein.
The immunotherapy pipeline is led by an in-licensed EP4 antagonist that Ikena is testing combined with Keytruda in phase 1b/2 colorectal and non-small cell lung cancer clinical trials. Ribometrix has presented that many RNAs implicated in disease comprise structural pockets amenable to targeting with small molecules.
The US biotech has just reported phase 2 trial results with the small, interfering RNA (siRNA) gene-silencing drug showing that it an cause a 90% or greater reduction in Lp(a) levels – a risk factor for cardiovascular disease – that was sustained over 48 weeks of follow-up.
The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Oxlumo (lumasiran) was also approved in the European Union last week, making it the third from Alnylam’s pipeline of RNA interference therapeutics to make it to market.
Sanofi is betting the genetic technology behind the fast development of two highly effective coronavirus shots last year will result in vaccines for other viruses as well as drugs for diseases of the lung and liver, announcing a deal to acquire a research partner Translate Bio for USD 3.2 Sanofi bets USD 3.2 No financial terms were revealed.
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.
The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. . Laying down a new track for RNA processing, Remix launched with $81 million in financing. The impact of CNS diseases extends beyond patients—to their families and society as well.”
Y-mAbs Therapeutics has a target action date of November 30 for its Biologics License Application (BLA) for Danyelza (naxitamab) for patients with relapsed/refractory high-risk neuroblastoma. The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs.
The bolt-on deal comes – which includes an upfront payment of $120 million and up to $910 million in milestones – covers rights to ARO-HSD, Arrowhead’s RNA interference drug targeting an enzyme called HSD17B13 that seems to contribute to the pathology of NASH and other liver diseases.
In the ATLAS study, CABENUVA met the primary endpoint for noninferiority (the proportion of participants with plasma HIV-1 RNA ?50 50 copies per milliliter [c/mL] at Week 48), with a comparable number of patients receiving either CABENUVA or their daily current antiretroviral regimen (CAR) having an HIV-1 RNA level ?50
As the first and only small interfering RNA to provide effective and sustained LDL-C reduction, Leqvio helps manage a critical cardiovascular risk factor for ASCVD. Leqvio is the first and only approved small interfering RNA (siRNA) LDL-C-lowering treatment in Europe 6,7. It is currently under review by the U.S. a statin or ezetimibe).
Dravet syndrome is most commonly caused by a genetic mutation in the SCN1A gene and affects approximately 1 in 15,000 to 1 in 21,000 people in the United States. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetic and Hematology, Neuroscience, and Gastroenterology (GI).
About Moderna Moderna is advancing messenger RNA (mRNA) science to create a new class of transformative medicines for patients. For more information, visit www.merck.com and connect with us on Twitter , Facebook , Instagram , YouTube and LinkedIn. For more information, visit www.novavax.com and connect with us on Twitter and LinkedIn.
is advancing messenger RNA (mRNA) science to create a new class of transformative medicines for patients. For more information, visit. www.merck.com. and connect with us on. For more information, visit. www.novavax.com. and connect with us on.
In 2010, the Association for Molecular Pathology (AMP), among other organizations, brought suit against Myriad Genetics, challenging the validity of the company’s patents on the BRCA 1/2 genes. animal, viral, bacterial) DNA and RNA sequences. Supporters of AMP v.
In hereditary angioedema (HAE), for example, donidalorsen an investigational RNA-targeted prophylactic treatment has demonstrated impressive efficacy. The FDA has accepted a Biologics License Application (BLA) and designated Priority Review to UX111, offering a potential option for affected families.
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