Biotech startup Typewriter Therapeutics has emerged from stealth with $56m in capital as it looks to progress its non-viral in vivo gene therapy platform closer to the clinic.
The Series A funding round, which was led by big industry hitters RA Capital Management and AN Venture Partners, will help push its target-primed reverse transcription (TPRT) technology platform closer to the clinic.
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To begin with, Typewriter will focus on progressing the TPRT platform by focusing on its in vivo CAR-T and hereditary liver diseases, which the company touts as “high-value indications with life-changing potential to patients”. With the funding from this round, the biotech plans to initiate non-human primate studies on the platform at the latter end of 2026, while establishing its first in vivo CAR-T development candidate.
Typewriter has designed its re-dosable, non-viral TRPT platform to harness the R2 retrotransposon – a natural system that allows for the insertion of a therapeutic gene into a specific target site. The RNA-based system is delivered via lipid nanoparticle (LNP).
Non-viral approach gains traction amid viral safety bottlenecks
Typewriter debuts its TPRT technology as cell and gene therapy (CGT) experts increasingly recognise the promise of non-viral approaches to diminish the high manufacturing cost and safety concerns associated with viral-based approaches.
Recently, Novartis hit pause on the development of its lentiviral-based CAR-T therapy, rapcabtagene autoleucel (rap-cel; YTB323) in several autoimmune diseases after three patient deaths linked to immune-related adverse events (AEs).
Similarly, Bristol Myers Squibb (BMS) paused enrolment into its autoimmune trials investigating lentiviral CAR-T, BMS-986353, due to “transient and reversible inflammatory events”, William Blair analysts declared in a research note.
There have also been several deaths in gene therapy trials from Sarepta, Pfizer, Intellia, Rocket and Capsida – leading to some in the space reassessing their gene delivery strategies.
According to experts interviewed by Pharmaceutical Technology, non-viral approaches could represent the future direction of gene therapy, but most don’t currently expect them to displace the viral vector-based methods in the near term.
On the cell therapy side, in vivo CAR-Ts are garnering significant attention for their potential to simplify the administration process and mitigate manufacturing challenges linked to their ex vivo counterparts. In recent years, several big pharma players have hedged their bets on the modality, with companies like Johnson & Johnson (J&J), Eli Lilly, AstraZeneca and Sanofi inking high-profile deals in the space.
While some are touting in vivo CAR-Ts as the next frontier in cell therapies, experts previously told Pharmaceutical Technology that suppliers will need to tackle the technical and logistical barriers to commercial viability before this drug class can see true success.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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