Developing curative gene editing therapies for genetic diseases with high unmet medical need
Massachusetts-based stealth mode biotechnology company focused on developing curative gene editing therapies
Discovered and characterized novel Cas9 variants with distinct advantages over traditional spyCas9
Combining optimized CRISPR Cas9 variants with modular tissue-specific delivery strategies
Expanding the capabilities of CRISPR gene editing for precise disease targeting
Bioinformatic pipeline to identify novel nucleases and sgRNA
Biochemical assays to determine PAM sequences for novel nucleases
AI-assisted optimization of nuclease activity and PAM sequences
Tissue-specific delivery strategies teilored for targeted organ systems
Focusing on diseases with high unmet medical need and clear genetic targets
Lead Program (Target Undisclosed): US$ 150B (2024 est) market opportunity across all indications expected to grow to US$ 250B by 2033.
Target undisclosed: US$ 90B (2024 est) total market opportunity across all indications expected to grow to US$ 155B by 2033.
Multiple Undisclosed Targets: US$ 16B (2024 est) total market opportunity across all indications expected to grow to US$ 45B by 2033.
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