Gene Editing — Now FDA-blessed.

In December 2023, the CRISPR/Cas9 gene editing therapy, known as exa-cel, received FDA approval. Developed collaboratively by CRISPR Therapeutics (Zug, Switzerland) and Vertex Pharmaceuticals (Boston, USA), this treatment targets a gene crucial for maintaining the function and disk-like shape of red blood cells. Specifically designed to treat sickle cell disease, exa-cel’s approval by the US FDA, following its earlier endorsement by the MHRA in the UK, represents a significant regulatory advancement in the treatment of genetic disorders.

Our gene editing industry report presents a snapshot of the current landscape and the promising future of this transformative field.

Key Highlights:

1. The top-5 publicly traded CRISPR companies — Intellia, CRISPR Therapeutics, Editas, Beam, and Prime Medicine — have displayed varied market cap dynamics since early 2023. Notably, CRISPR Therapeutics and Editas grew by 1.5x and 1.3x, respectively, while Prime Medicine experienced an almost 2x decline.
2. 26 gene editing clinical trials remain active predominantly focusing on ex vivo strategies (20 out of 26) and blood indications (18 out of 26).
3. Editas appears to have struggled with its AAV-based therapy trials, in contrast to Intellia, which leads the in vivo editing field with 3 out of 6 trials ongoing.
4. Feng Zhang and MIT are likely ahead in the gene editing patent war, overshadowing Nobel Prize winners Jennifer Doudna and Emmanuelle Charpentier, while contributions from Virginijus Šikšnys remain less known.
5. Large insertions and epigenetic editing are gaining prominence, evidenced by eight VC rounds of over $50M and notable academic research.
6. Fanzor, emerging from Feng Zhang’s lab, is poised to become a rival to CRISPR/Cas technology, reigniting discussions about novel gene editors of eukaryotic origin.
7. The non-viral, non-liver delivery remains the challenge — while ReCode Therapeutics being the only company in clinics with lung-targeting SORT LNPs, 89% of the 3900+ gene therapies in development still rely on AAV-based methods.

Please refer to our full report.

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