In the rest of this post I will discuss what those opportunities are, the potential market opportunity, and why I suspect that investors are better off buying into this $4.5bn market cap company than any other publicly traded CRISPR-focused company at the present time. The reality is that with it being more than two years since Doudna and Charpentier's Nobel Prize, and 10 years before scientists began investigating CRISPR, these companies stock prices can no longer trade on hype and the possibility of breakthrough, "one and done" therapies, and need to start showing they can win approvals for drugs that genuinely work.Īt this time there's only one company close to achieving this market validation and that's Crispr Therapeutics. Editas Medicine ( EDIT), founded by scientists at the Broad Institute, Harvard, who have patent disputes ongoing with Doudna and Charpentier, and the University of California at Berkeley and the University of Vienna (collectively known as CVC) over who adapted the technology to treat human disease first - has seen its share price fall by 55% over the past five years.Ĭaribou Biosciences ( CRBU) - another CVC vehicle - has seen its stock fall by 44% since its July 2021 IPO raised ~$304m, while the Broad Institute's next generation CRISPR / base editing vehicle Beam Therapeutics ( BEAM) has been a success, up 47% since its Feb 2020 IPO raised $180m. Some other CRISPR focused companies have admittedly not fared so well.
These 2 companies share prices are respectively up 146%, and 210% over the past 5 years, versus the S&P 500's 53% gain over the same period. This discovery spawned several biotech companies - Doudna became a scientific co-founder of Intellia Therapeutics ( NTLA) whilst Charpentier co-founded CRISPR Therapeutics ( NASDAQ: CRSP) - the subject of this post. In nature, CRISPR is used by bacteria to identify genetic sequences belonging to harmful viruses and cleave them using specialised enzymes, such as CAS-9, and Doudna and Charpentier were able to show that this system could be adapted to target and edit, remove, or destroy harmful genetic sequences in humans. CRISPR stands for Clustered, Regularly Interspaced Short Palindromic Repeats, and its discovery earned the scientists Jenifer Doudna and Emannualle Charpentier the Nobel Prize for Chemistry in 2020. There's one field of gene therapy research that has rewarded investors handsomely over the past five years, however, and that's CRISPR gene editing. Sio Gene Therapies ( OTC:SIOX) stock is down 82% in the past 12 months, Generation Bio ( GBIO) is down 69%, and Voyager Therapeutics ( VYGR) stock is down 63% across the past five years. Within five years, the drug becomes more expensive than Zolgensma.ĭeveloping gene therapies is fraught with complex and difficult challenges, however, and most listed companies that have attempted to do so have ended up burning through investors cash in the clinic, without producing a drug that works. Spinraza, administered four times per year, every year (since it is not a "functional cure") costs $750k for the first year of treatment and $350k per annum after that. Compared to Biogen's ( BIIB) Spinraza, also indicated for SMA, however, Zolgensma can be considered cheap. Novartis' ( NVS) Zolgensma, for example, which is indicated to treat Spinal Muscular Atrophy ("SMA") in infants, comes with a >$2m price tag. That is also why approved gene therapies tend to be the most expensive on the market. a "one and done therapy," that after being administered a single time, cures the patient for life.
The reason gene therapy is such an exciting field of research is that it offers the possibility of "functional cures" i.e. Despite these breakthrough successes, Bluebird's share price has sunk in value by >90% across the past five years. Gene therapy is one of the most exciting and innovative fields of drug development research, although for biotech investors it's also one of the most risky places to park your money.Ĭonsider Bluebird Bio ( BLUE) for example, a company that won not one, but two approvals for gene therapies - Skysona, approved for the rare disease cerebral adrenoleukodystrophy ("CALD"), and Zynteglo, for the blood disorder beta thalassemia.
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