Shape Therapeutics, Inc. (ShapeTx), has received $35.5M in Series A financing, led by New Enterprise Associates (NEA), with additional participation from CureDuchenne Ventures. The new capital will enable the company to extend its growing portfolio of intellectual property, recruit and hire top scientific talent and advance its groundbreaking RNA and protein targeting platforms focused on curing human diseases.

These platforms include the proprietary ShapeTx RNAfix technology that enables direct in vivo targeting and modification of RNA by leveraging proteins such as Adenosine Deaminases Acting on RNA (ADARs), suppressor tRNAs, and engineered adeno-associated viruses (AAVs). The RNAfix platform differentiates from other contemporary genome engineering technologies by engaging natural human cellular machinery to modify RNA.

ShapeTx was founded on the work of Dr. Prashant Mali, Assistant Professor of Bioengineering at UCSD, who during his postdoctoral fellowship in the George Church laboratory at Harvard Medical School pioneered the use of CRISPR in human cells. ShapeTx RNAfix platform is built upon his lab's most recent work demonstrating in vivo use of guide RNAs to recruit native ADARs and to fix mutations in multiple rare genetic disease mouse models.

Our technology can correct mutations or target specific genes in neurodegenerative, oncology, metabolic and rare genetic disorders by hijacking naturally occurring proteins such as ADARs present in our cells using just a short guide RNA. Our proprietary new platform avoids the risk of in vivo immunogenicity and permanent off-target damages commonly associated with CRISPR-based approaches, explained Francois Vigneault, Ph.D., President and CEO, who was previously VP of Research at Juno Therapeutics after a successful co-acquisition of AbVitro, Inc. by Juno and Celgene.

Ed Mathers, Partner at NEA and Board member at ShapeTx, said, One rarely comes across a proprietary technology platform with such transformative potential led by a focused and data-driven scientific group with a successful track record in pre-clinical and clinical development. 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. NEA looks forward to backing the company in future rounds as they move the technology toward the clinic.

While the ShapeTx platform will be enabling for many other genetic diseases, Dr. Mali's...

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