Compared to the smallest mRNA molecule, which is 300 nucleotides long, the largest transfer RNAs (tRNA) is less than a third of the size at 76 nucleotides. Scientists at Alltrna are harnessing the unique biology of tRNAs to engineer a single tRNA medicine that could treat thousands of rare diseases that share the same genetic […]
Roche has announced a partnership with Alnylam to develop and commercialize zilebesiran, an investigational RNAi therapeutic currently in phase 2 for the treatment of hypertension. Hypertension affects more than 1.2 billion adults worldwide and is the main, independent, preventable risk factor for death and disability, with approximately 80% of the adults with hypertension worldwide not […]
Korro Bio, Inc., an RNA editing company focused on the discovery and development of novel genetic medicines, and Frequency Therapeutics, Inc. have entered into a definitive merger agreement to combine the companies in an all-stock transaction. The combined company will focus on the advancement of Korro Bio’s portfolio of RNA editing programs, is expected to […]
By Ashish Dhir, PhD and Amy Walker, PhD, 4basebio The pace, efficacy and scalability with which the success of mRNA vaccines against COVID-19 was demonstrated opened endless possibilities for mRNA medicine in broad areas of infectious diseases, cancers, protein-encoding replacement therapies. The advantages of RNA medicine are obvious, with the ease and speed of design […]
Flamingo Therapeutics has been awarded a research grant of €1.7 million ($1.9 million) by Flanders Innovation & Entrepreneurship (VLAIO). The award represents the funding at 60% of a €2.9 million ($3.2 million) project. Funding from the grant will be used to advance Flamingo Therapeutics’ RNA-targeting oncology portfolio, including translational research for its lead clinical program, […]
Rznomics Inc., a South Korea based biopharmaceutical company specializing in the development of RNA-based gene therapeutics, has received phase 1/2a IND approval from the U.S. FDA for its glioblastoma multiforme (GBM) treatment. RZ-001 initially obtained IND approval for hepatocellular carcinoma (HCC), but Rznomics also found pre-clinical efficacy in GBM models and submitted the IND for […]
SiSaf Ltd, an RNA delivery and therapeutics company, has announced that SIS-101-ADO, its siRNA therapeutic for patients with autosomal dominant osteopetrosis Type 2 (ADO2), has been granted orphan drug designation by the U.S. FDA. Also, due to the serious manifestations of this rare skeletal disorder in children, SIS-101-ADO has been granted rare pediatric disease designation […]
Exothera has partnered with Quantoom Biosciences to gain access to Quantoom’s Nfinity technology, a continuous production platform for RNA. The partnership will enable Exothera to be the first CDMO in the world to offer this off-the-shelf process for the continuous production of RNA as a service. Continuous manufacturing of RNA can provide significant advantages in […]
Arrowhead Pharmaceuticals Inc. has announced interim results from an ongoing phase 1/2 clinical study of ARO-RAGE, an investigational RNA interference (RNAi) therapeutic designed to reduce production of the receptor for advanced glycation end products (RAGE) as a potential treatment for inflammatory pulmonary diseases, such as asthma. These data represent the first clinical demonstration of the […]
Our genetic code includes more than 15,000 specific sections that can be made into molecules called lincRNAs. Some of these sections can occur in coiled-up sections of our genome called topologically associating domains, or TADs. LincRNAs derived from TADs appear to act as markers indicating the specific kind of tissue they are within. When something […]
Researchers from Tel Aviv University in Israel have revealed details of a new approach to the treatment of ovarian cancer using RNA-based nanodrugs – demonstrating an 80% survival rate in lab models.In a study at Tel Aviv University, the protein CKAP5 (cytoskeleton-associated protein) was used for the first time as a therapeutic target for RNA-based […]