Pair of RNA start-ups pull in over $300 million in funding

09 Aug 2023
OligonucleotidesiRNA
Two companies working on advancing RNA medicines announced Wednesday that they have raised a combined $309 million in separate funding rounds. ADARx Pharmaceuticals, which took in $200 million in Series C investments, is looking to develop two next-generation RNA programmes, while Flagship-backed Alltrna said it raised $109 million in a Series B round to put toward a machine learning-driven platform based on transfer RNA (tRNA) biology.
Ricky Sun, a partner at Bain Capital Life Sciences, which co-led the round for ADARx with TCGX, said the company has developed "a unique siRNA platform that we believe has the potential to generate differentiated clinical data from several programmes over the near- and long-term."
ADARx says it will use the new funds to advance its ADX-324 programme for hereditary angioedema, as well as its ADX-038 asset under development for multiple complement-mediated diseases. It added that the fundraise, which also drew support from Lilly Asia Ventures, OrbiMed and SR One Capital Management as well as a string of new backers, will help finance a pipeline of mRNA silencing or editing candidates to treat a range of diseases.
The company raised $46 million in a Series B-1 round earlier this year, coinciding with the entry of ADX-324 into Phase I. According to CEO Zhen Li, early clinical data "strongly support the potential of ADX-324 to be the best-in-class treatment for patients suffering from hereditary angioedema," and also validate the start-up's targeted delivery PLR platform and SPE technology.
Meanwhile, Alltrna said its fundraising haul will help unlock the "combinatorial power of tRNA sequences and modifications," and move its first drug candidates towards the clinic for so-called stop codon diseases, an umbrella term that encompass thousands of genetic illnesses caused by a premature termination codon (PTC) mutation. Aside from Flagship, Alltrna said other participants in the round included "a number of top-tier investors."
"Since launch, we've gained unparalleled insights into tRNA biology and shown that we can systematically design and deliver tRNAs with universal readthrough of PTCs and restore full-length functional protein production in vivo," remarked CEO Michelle Werner. She noted that the company's platform "optimises tRNA nucleotide sequence and modifications to turn tRNA's sophisticated biology into programmable medicines with powerful potential therapeutic properties."
FirstWord recently caught up with Werner at the American Society of Gene and Cell Therapy (ASGCT) meeting where Alltrna presented some early data showing that its platform could deliver engineered, modified tRNA oligonucleotides with "significantly increased potency." You can read that interview here: ViewPoints: How Alltrna is leveraging tRNA biology to address genetic diseases.
For an overview of the RNA landscape, check out Spotlight On: The growing universe of RNA editing technologies and Spotlight On: Startups advancing tRNA therapeutics.
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