5 RNA editing companies you should know about

RNA editing companies

As patients were dosed in the first-ever RNA editing clinical trial in December 2023, it certainly seems as though RNA editing technology has started to make significant strides. Unlike DNA editing, RNA editing allows scientists to make changes in the molecules that carry the instructions needed to produce proteins, without actually changing the original DNA code, meaning that it could be a safer alternative to traditional gene editing techniques. More and more companies are now seeing promise in this type of technology and are diving into the field. In this article, we take a look at five companies leading innovation in RNA editing.

Table of contents

    AIRNA

    RNA editing company AIRNA launched in September last year, emerging from stealth with a $30 million initial financing. The company is pioneering the discovery and development of RNA editing therapeutics to deliver on the promise of genetically defined medicines for patients with both rare and common diseases. It is currently working on advancing its first therapeutic program, a best-in-class product candidate to treat the inherited genetic disease alpha-1 antitrypsin deficiency (AATD), as well as a pipeline of therapeutic candidates to address multiple diseases with high unmet need.

    ADAR-directed RNA editing delivers a safe oligonucleotide that is programmed to recruit an endogenous cellular enzyme, ADAR, to introduce a precise modification to a patient’s RNA, resulting in changes to the encoded therapeutic proteins. The company’s founders were the first to publish peer-reviewed papers showing ADAR-mediated RNA editing and the use of oligonucleotides (short, single, or double-stranded DNA or RNA molecules that have a wide range of applications in genetic testing, research, and forensics) to recruit endogenous ADAR for targeted RNA editing. 

    AIRNA’s therapies are driven by its RNA editing platform, called RESTORE+, which is a systematic advancement of this research that generates a pipeline of products by optimizing each therapy’s sequence, chemistry, and delivery for precise, efficient, and safe RNA editing. 

    Ascidian Therapeutics

    Ascidian Therapeutics is uniquely working on editing exons at the RNA level. Its therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With discovery, preclinical, and clinical programs in retinal, neurological, neuromuscular, and genetically defined diseases, Ascidian’s approach has the potential to treat patients with one dose of an RNA exon editor, meaning it could offer a cure to patients. 

    Ascidian’s current lead candidate is called ACDN-01 and is the only clinical-stage therapy targeting the genetic cause of Stargardt disease, which is a rare genetic eye disease that happens when fatty material builds up on the macula, causing vision loss. The disease itself is caused by 1,000 mutations across a gene called ABCA4. Diseases caused by defaults in the ABCA4 gene cannot be addressed by standard gene replacement because of how big it is, which is why RNA editing could be the best hope for correcting the genetic mutations responsible for these types of diseases.

    When the U.S. Food and Drug Administration (FDA) gave ACDN-01 the go-ahead to enter clinical trials in January, it became the first RNA candidate to enter in-human testing in the U.S. At the same time, the FDA also granted fast track designation for ACDN-01. 

    Korro Bio

    Korro Bio is an RNA editing company focused on the discovery and development of a new class of precision genetic medicines for both rare and highly prevalent diseases. Korro’s approach involves ADAR editing and it has a platform called OPERA, which it says is designed to solve many of the challenges facing current gene therapy and gene editing approaches, including safety and flexibility of delivery mechanisms. 

    The OPERA platform enables a breadth of indications with an initial focus on six potential programs. The company’s lead program is focused on treating alpha-1 antitrypsin deficiency (AATD), which is an inherited disease that results from a single genetic defect that manifests itself as a broad spectrum of clinical pathologies. The company is rapidly advancing this program into a clinical trial and intends to submit a regulatory filing in the second half of 2024. 

    In July last year, Korro Bio and regenerative medicine company Frequency Therapeutics announced that they had entered into a definitive merger agreement to combine the two companies, with a focus on advancing Korro’s RNA editing programs. The combined company is expected to have cash balance of approximately $170 million, which is expected to provide cash runway through several milestones and into 2026. 

    Wave Life Sciences 

    Wave Life Sciences is committed to developing best-in-class therapies across multiple therapeutic modalities using PRISM, the company’s proprietary discovery and drug development platform that enables the precise design, optimization, and production of stereopure oligonucleotides.

    In September 2023, Wave Life Sciences submitted a clinical trial application for its lead RNA editing candidate, WVE-006, and dosing for that trial has already begun in the U.K. This was a milestone achievement, as it marked the first time an RNA editing candidate managed to reach the clinic. WVE-006 is a synthetic RNA molecule that once again uses ADAR to edit RNA and is intended as a potential treatment for AATD. The therapy aims to correct the single base mutation in messenger RNA (mRNA) coded by the SERPINA1 Z allele, enabling the restoration and circulation of functional M-AAT protein in order to prevent liver damage and protect the lungs. 

    The initiation of the trial for WVE-006 saw Wave achieve its first milestone in its collaboration with GSK, which resulted in a $20 million payment to the RNA editing company. As part of the collaboration, Wave is eligible to receive up to $505 million in additional development, launch, and sales-related milestone payments, as well as tiered royalties on net sales.

    ShapeTX

    Shape TX is pioneering programmable RNA medicines to repair the genetic causes of disease and merges innovations in artificial intelligence (AI) and RNA technology to create programmable RNA medicine aimed at making transformative treatments more accessible to individuals globally. 

    The RNA editing company’s platform enables pharma innovators to design treatments across a wide range of diseases, including rare genetic disorders as well as debilitating conditions, such as Alzheimer’s, Parkinson’s, and many more.

    In August 2021, Shape entered into a strategic research collaboration with Roche to advance breakthrough AAV-based RNA editing technology for neuroscience and rare disease indications. This collaboration was then expanded in November last year, in which the companies added a new target to the collaboration in which Shape will use its RNA editing platform to develop a potential one-time therapy for patients with high unmet needs for an undisclosed disease affecting millions of people worldwide. Additionally, Shape entered into another collaboration with Otsuka last year, to develop novel AAV therapies for ocular diseases.

    RNA editing market set for growth

    According to TechSci Research, the global RNA editing technologies market was valued at $261.28 million in 2023, and is anticipated to project impressive growth, with a compound annual growth rate (CAGR) of 6.94% through 2029. The market is driven by increasing research investments, a growing demand for personalized medicine, and the expanding applications of RNA editing technologies across biotechnology. Therefore, it is highly likely that we will begin to see more and more companies entering the RNA editing space in the near future.

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