The FDA just approved the first drug, Luxterna, to cure a rare form of genetic blindness by changing DNA. It's not the first gene therapy ever approved (it's third), but it is the first time the FDA has ever approved an injected drug that changes the inherited DNA of a person's cells to effect a cure.
FDA Commissioner Scott Gottlieb recently remarked, regarding gene therapy, "we're at the early stages of a transformation in medical treatment as a consequence of this new technology. And the benefits are likely to accelerate quickly."
Earlier this year was the first time a hospital biohacked a patient to try to cure a genetic disease. While it takes time for gene therapies to be tested, trialed, and possibly approved by the FDA, genetics startups like Spark, AveXis, Caribou Biosciences, Editas, and Audentes have been on the road a while. MIT estimates over 40 new therapies would be approved by FDA in the near future.
New breed of startup tackles genetics
A new breed of software company is developing for the genetics industry. Just this year, over 50 U.S. genetics and gene therapy startups raised at least $1 million to support genetics-based treatments, including speeding trails, improving accuracy of tests, and providing better platforms. Collectively, this new kind of startup can help drive down the cost of gene therapy discovery for patients.
Here are some of the startups to watch in 2018 that back discoveries in genetics and gene therapy.
Synthego, founded by former SpaceX engineers, aims to be the 'AutoCad of gene editing.' This month, it just released its first gene knock out kit, an application it has been developing with major research institutions like Salk Institute and St. Judes.
"A constant challenge for our research is trying to reduce the number of clones we need to screen to find a desired targeted modification. In our tests, the Gene Knockout Kit gave us greater than 80 percent knock-out rates for seven targets," shares Shondra Miller, Ph.D., Director, Center for Advanced Genome Engineering at St. Jude Children's Research Hospital, on Synthego's web site. The company offers researchers a money-back guarantee you'll get 50 percent or better editing in any human cell type for research use.
Paul Dabrowski, CEO and co-founder of Synthego, shared with me in email that, "It's possible to imagine a day when gene therapies are safe, effective, easy and affordable for anyone who needs them. Right now, the first CRISPR gene therapies are in development, but they are not built on a 'standard stack' and require a lot of custom work for each indication - these therapies will be coming into the clinic and hopefully get approved the coming couple years, and after that there could be significantly more investment into standard tools."
Edico, Backed by investors like Dell, Qualcomm and others, San Diego-based Edico is the backend for bioinformatics. Their DRAGEN data platform offers a dedicated processing environment tuned to the massive data analysis needed for gene research. According to the company, "DNA sequencing technology is advancing at an even more rapid pace than the cell phone revolution. By increasing the speed and accuracy for NGS data analysis like whole genome sequencing (WGS), our computing platform makes it easier to discover links between DNA sequence variations and human disease."
Trace Genomics, founded in 2015 by Diane Wu and Poornima Parameswaran in San Francisco, has been discussed as the 23&Me of dirt. The agtech startup has raised $4 million in one round to test farm soil for its microbiome. An initial screening cost of only $199 allows farmers to have their soil samples analyzed by Trace, which applies machine learning to uncover not just what's in there, but what it means for crop yield and productivity.
Paradigm Diagnostics of Pheonix, AZ offers a gene-specific cancer test that helps physicians pinpoint the best cancer therapies for their patients based on DNA analysis. It just raised a $7 million Series B.
There are a number of software startups focused on improving cancer treatment with better analysis, like early stage CureMatch, based in San Diego. "Nothing could be more important than precisely identifying DNA code alterations that drive the cancers' growth and use that information to decide on treatment plan," Dr. Stephane Richard, CEO, shared with me in email.
Celmatix. Based in New York, Celmatix just earned $4.5 million dollar grant from the New York Economic Development Council for expanding in Manhattan. The company has developed a software-driven testing platform that helps families and physicians make fertility choices faster and more accurately for individuals. "Women can now make potentially life-defining decisions about how to proactively plan for the family they want to build and be more efficient in overcoming fertility difficulties they are experiencing using better, more personal, information than age," founder Piraye Beim shared earlier this year in TechCrunch.
Genoox, an Israeli startup now based in Palo Alto, is a cloud research platform for sifting massive medical trial data quickly and accurately. Their website says, "Simplify complex sequencing data and make impactful discoveries using the most advanced genetic analysis tools and applications."
Insilico Medicine, originally a Latvian startup now based in Baltimore, develops custom AI learning platforms that uncover insights based on biology. Their latest product is Chemistry.AI.
23&Me. No list of major gene therapy movers would be complete without 23&Me. At one time blocked by the FDA, 23&Me is now a smash hit with both consumers and pharma companies. Its kit was one of the top 10 sellers on Amazon's Prime Day this year. Plus, its treasure trove of DNA sample data from millions of customers is just the kind of testing data many of the next generation of gene therapies need.
Founder Anne Wojcicki recently expressed her sense of hope in the future of genetics to the New York Times, "Genetics is like your cholesterol test. So your cholesterol test is going to tell you if you have high cholesterol, which is a risk factor for heart disease. But it's not saying you're going to die immediately from heart disease or even die at all from heart disease. It's just saying you have a risk factor. And so genetics is similar. It's saying you have a risk factor. So the beauty to me of genetics is, it's always a story of hope."