Resolves YES if by January 1, 2025, any of the following conditions are met:
5 or more gene-editing curative therapies have successfully completed phase III clinical trials for treating at least one major disease;
A gene-editing curative therapy is approved by the FDA or the EMA for treating at least one major disease in the US or Europe;
A gene-editing curative therapy has been endorsed by the WHO as an essential medicine for treating at least one major disease globally.
The above may be limited in scope: for example, in a breakthrough therapy FDA approval, or an approval for a subset of patients. Pre-approval access (e.g. compassionate use) is not counted.
Resolves NO otherwise.
Definitions:
A major disease is defined by inclusion in the Global Burden of Disease study (most recent version available by 2025; excluding injuries; with appropriate translation between GBD and other classifications).
A gene-editing curative therapy is defined as a technique that introduces targeted and precise changes in the DNA sequence of living cells (such as CRISPR or TALEN) and that cures or prevents the disease.
Curing or preventing the disease is defined by such language – including close synonyms – being endorsed by at least one of the FDA, EMA, WHO, CDC, NIH, or NICE, regardless of scope, channel, and minor qualifications (ie. it may be considered a cure only for a subset of patients; it may be a press release; it can be a “promising” cure, etc.)
Successful completion of phase III clinical trial means that the therapy is reported to have met its primary endpoint(s) with planned statistical thresholds and has demonstrated an acceptable safety profile.
The market is based on Sam Altman’s tweet from January 2019: “Predictions for the three most important technological developments that will happen by 2025: (…) Gene editing will have cured at least one major disease” (https://twitter.com/sama/status/1081584255510155264).
Related questions
I think recent FDA approvals of Casgevy and Lyfgenia might count.
The FDA does not use the word "cure", but the outcomes are pretty stunning and the targeted endpoints ("The primary efficacy outcome was freedom from severe VOC episodes for at least 12 consecutive months during the 24-month follow-up period", "Effectiveness was evaluated based on complete resolution of VOEs (VOE-CR) between 6 and 18 months after infusion with Lyfgenia") sound a lot like "promising cure" to me in practice.
CNN and NBC both use the word "cure", with more or fewer qualifications.
The Casgevy press release says it "offers the potential of a functional cure". The Lyfgenia press release doesn't use the word cure: "for the treatment of sickle cell disease ... has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease."
Sickle Cell is one of the disease on the Global Burden of Disease list. (Wiki link.)
@EvanDaniel regardless of what the market creator decides, this is pretty amazing news =)
@PeterBuyukliev Agreed, I read this and was genuinely a bit shocked that we are already there, it is amazing. Sad that one treatment is 2.2 mil though :")
can you define "major disease"? Does this include rare diseases? Those are the prime targets for gene therapies atm.