Google’s parent company, Alphabet, is now pouring funds into a start-up wants to gene-edit heart disease out of existence using a one-time injection.
Verve Therapeutics hopes to leverage its CEO’s discovery of a gene he believes is linked to lower heart disease risks to ‘fix’ the DNA of people at high risk of heart disease.
And the company plans to use CRISPR – the costly, controversial gene editing tool used by Chinese scientists to rid twin girls of HIV in the womb – to accomplish it.
Verve plans to inject people with CRISPR carrying a copy of the protective gene, in theory cutting their heart disease risks by up to 80 percent.
It’s a bold and novel plan, but one that could do more to widen already-gaping disparities in heart disease or tackle basic risks like obesity.
Google’s parent company, Apple, is helping to fund Verve Therapeutics, a start-up that wants to make a single gene-editing injection to protect people against heart disease
One in four deaths in the US each year are due to heart disease.
It remains the leading cause of death, costing American taxpayers $200 billion a year.
Now, Google’s parent company Alphabet, is throwing some private money at deadliest disease in the country.
Alphabet got into the venture capital and health technology games through GV and Verily, respectively, NBC reported.
GV invests in companies, startups and other ventures that its umbrella company sees potential in, while Verily, a now-independent company that got its start in Alphabet’s incubator, tackles a diverse smattering of health research projects.
Verily rounded up an additional $1 billion in external funding last year, some of which will go to its partnership with Verve, while GV added the better part of another $60 million in funding to the pot shared by Verily and Verve last week.
To Verve’s efforts, Verily plans to add its research on nanoparticles, which it believes will provide the best system by which to deliver Verve’s gene-editing vaccine.
Nanoparticles are still in their early days, but because their small size allows them to enter hard-to-access tissues undetected by the immune system and deliver extended release treatments there.
Now, Verily wants to use them to help Verve deploy CRISPR to the DNA of heart disease-prone patients.
For its part, Verve has made it its mission to ‘safely edit the adult genome and confer lifelong protection from cardiovascular disease.’
The basis for its moonshot effort is CEO Dr Sekar Kathiresan’s – formerly a geneticist at Massachusetts General Hospital – research on a gene called PCSK9.
PCSK9 plays an important role in the regulation of blood cholesterol, and certain mutations of the gene cause a form of high cholesterol called hypercholesterolemia, which is passed down in families.
Other variants of the gene, however, seem to have protective effects, and people with these copies have lower cholesterol, fewer heart attacks and good overall health, Verve claims.
One study on PCSK9, done in 2006, found that black people – who generally have higher risks of heart disease – who had ‘nonsense mutations’ in the gene had 28 percent lower cholesterol and 88 percent lower risk of heart disease.
White people with the same mutation had a 47 percent reduction in their heart disease risk.
CRISPR edits genes by finding a target segment of DNA, ‘snipping’ out copies variants that create diseases or disease risks and replacing them with a new bit of DNA.
‘Verve aims to develop therapies, administered as a single dose, that mimic naturally protective variants to confer lifelong protection against coronary artery disease in adults,’ the company explains on its website.
The company says its goal is to replace the daily cocktail of pills many have to take to keep heart disease at bay.
It sounds like a simple, elegant and lasting solution.
But gene-editing is anything but – and we don’t even know what the potential fallout would be.
CRISPR is still in its nascent clinical stages. Although scientists suggest therapeutics that use it could be made at $65 dose, that doesn’t include the research needed to design the ‘vaccine’ Verve promises.
And we have only begun to understand the complex network through which genetic and environmental risk factors interact.
A protective gene, for example, might lessen the effects of obesity on cholesterol – but it won’t prevent obesity itself, a more complicated risk factor for the disease.
DNA itself is still a relatively opaque subject, and even in past year, we’ve discovered new cascades of consequences for genes that have already been edited.
For example, the gene that Chinese scientists edited in the pair of embryos – now twin girls, over a year old – in order to protect them from HIV they would’ve inherited from the father was discovered to alter brain function as well shortly after the girls were born.
We don’t yet know what other advantages – or disadvantages – the nonsense-mutated PCSK9 gene might confer to those Verve wants to inject with it in the years to come.