A company developing a means to edit RNA has raised more than $30 million in a venture capital funding round.
Seattle-based Shape Therapeutics, or ShapeTx, said Tuesday that it had raised a $35.5 million Series A financing, led by New Enterprise Associates and with participation from CureDuchenne Ventures. The company said it plans to use the money to build up its intellectual property, hire scientists and advance its RNA and protein-targeting technology, which it describes as being aimed at curing genetic diseases.
The company describes its technology, RNAfix, as allowing for direct in vivo targeting and modification of RNA by using engineered adeno-associated viruses, suppressor tRNAs and proteins like adenosine deaminases acting on RNA, or ADARs.
ShapeTx further said that RNAfix differentiates from other contemporary genome engineering technologies by using natural human cellular machinery to modify RNA. It says its system avoids the risk of in vivo immunogenicity and permanent off-target damage commonly associated with CRISPR/Cas9 gene-editing approaches, which as of late have grown to become the most common means of gene editing in the context of human diseases, with an increasing number of therapies in the clinic or moving toward it. The company’s website states that its technology has been validated in numerous in vivo disease models – i.e. in animal studies — though it has not tested them in the clinic.
“One rarely comes across a proprietary technology platform with such a transformative potential led by a focused and data-driven scientific group with a successful track record in pre-clinical and clinical development,” NEA partner Ed Mathers said in a statement. “The team has shown us an exciting demonstration of the technology in multiple in vivo models, alongside one of the strongest IP estates we have seen in the field.”
Other companies have also been exploring cleaner methods of gene editing beyond CRISPR/Cas9. Among these is Beam Therapeutics, which is developing base editing technology to enable genome editing at the single-letter level. Last week, it formed an agreement with Prime Medicine – founded by one of Beam’s own co-founders – to in-license a related technology called prime editing. Prime editing would enable correcting primary genetic causes of diseases – for example sickle cell disease or Tay-Sachs disease – without creating too many unwanted effects.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
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