A preliminary assessment of Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1) showed the first-ever targeted delivery of RNA into muscle, an area previously untreatable with existing RNA therapeutics. Sarah Boyce, Avidity’s CEO, said in a Dec. 14 call that the antibody oligonucleotide conjugate’s data were unprecedented in the RNA space and in myotonic dystrophy type 1 (DM1), labeling it a “revolutionary advancement.”
Rgenta Therapeutics Inc.’s $52 million in a series A money will let the RNA-focused firm pursue its small-molecule drug efforts “for the next two or three years,” as candidates in cancer and neurology make their ways toward the clinic, said co-founder and CEO Simon Xi. “We’ll go where the science leads us,” he told BioWorld, adding that the cash on hand is sufficient to complete a phase I study.
With its acquisition of Subintro Ltd., Rigimmune Inc. put a building block in place to further its development of stem-loop RNA therapeutics that selectively activate the innate immune sensor RIG-I. Subintro specializes in development and delivery of antiviral therapeutics for respiratory diseases caused by RNA viruses, including influenza, rhinovirus and SARS-CoV-2. Subintro’s technology allows the company to consider topical and nasal delivery.
The extraordinary proliferation of different genetic therapeutic modalities in the last decade has not been matched by a commensurate flourishing of delivery technologies. The liver is the natural destination for many carriers when administered systemically. But getting beyond the liver to other organ systems has proven to be a significant challenge. Only a tiny percentage of carriers – even those with targeting moieties – escape first pass metabolism in the liver and reach their target destination. “The key issue is getting out of the liver,” Nessan Bermingham, founder and interim CEO of Liberate Bio, Inc., told BioWorld. “That’s not a trivial problem.”
Two research teams independently reported in the Oct. 5, 2022, issues of Nature (Duke University) and Nature Biotechnology (Stanford University) on the development of RNA sensing technologies designed to target cell-specific RNA sequences to form a double-stranded RNA:RNA hybrid that is then edited by endogenous ADAR proteins to remove a stop codon and ultimately enabled to express any protein placed within the construct. The target design typically starts from single-cell RNA transcriptomics data that previously identified cell-specific RNA transcriptomics.
Skyhawk Therapeutics Inc. has cut a deal with Sanofi SA to discover and develop small molecules to treat targets in oncology and immunology, adding to its lengthy list of partnerships.
Remix Therapeutics Inc., a company developing small molecules to manipulate RNA processing, stands to earn upward of $1 billion through a new strategic collaboration with Janssen Pharmaceutica NV. A Cambridge, Mass.-based startup, Remix last year patented new RNA splicing modulators. It will receive an initial payment of $45 million for research funding plus potential preclinical, clinical, commercial and sales milestone payments, and royalties. Janssen gains exclusive rights to three targets with applications in immunology and oncology.
Infectious disease specialist Aicuris Anti-infective Cures AG has signed a worldwide license agreement worth up to €100 million (US$114 million) with Hybridize Therapeutics for a potential therapy to prevent BK virus (BKV) infections in immunocompromised patients.
HDT Bio Corp. looked at the world and saw health care inequity, so it built itself to help countries with developing economies help themselves. The company is bringing RNA technology for handling COVID-19 to underserved areas such as Brazil so they can develop, manufacture and distribute their own vaccine instead of relying on big pharma or developed-nation governments.
Ceptur Therapeutics Inc. has completed a $75 million series A financing to develop targeted oligonucleotide therapies based on its U1 adaptor technology. The adaptors are bivalent oligonucleotides designed to engage sequence-specific mRNA and the U1 small nuclear ribonuclear protein that regulates transcription and splicing. The therapeutics are for controlling gene expression at the pre-mRNA level within the nucleus.
