This week’s roundup includes several reads about a crucial but often unnoticed shift in the life sciences ecosystem—the interplay between the digital future of life science, the development of next generation therapeutics, and the promise of personalized medicine.
The drop in cost and increase in accuracy of genome sequencing has not only led to the rapid development of COVID-19 vaccines, but it has made at-home gene sequencing almost a novelty, as one of the articles below explains. We also take a look at how further advancements in life science technology and computing capabilities have led to effective CRISPR therapies and the use of artificial intelligence in drug discovery. These breakthroughs will revolutionize the future of medicine, but will also create new financially and morally complex issues when it comes to access and affordability.
Each week, we aggregate industry news and highlight five things you need to know about in the life sciences space. Here’s the latest.
One year later: Results among the first U.S. patients treated with CRISPR
The gene-editing technique known as CRISPR is talked about so much in the media and scientific circles that is hard to remember that only a handful of patients have actually been treated with it. Victoria Gray is one of those early patients who received a CRISPR therapy to treat her sickle cell disease. A year later, she and nine other patients from her treatment cohort are doing well, according to a recent report from the American Society of Hematology.
These results are a major step forward in understanding the safety and efficacy of CRISPR – which permanently edits a patient’s DNA – and other gene editing techniques that are on the cutting edge of personalized medicine. The promising result so far in Gray’s case “gives us great confidence that this can be a one-time therapy that can be a cure for life,” CRISPR Therapeutics CEO Samarth Kulkarni tells NPR in the piece linked above.
Primary care physicians further embrace at-home genetic tests
A recent study conducted by Medscape and DNA testing company 23andMe shows that primary care physicians are increasingly comfortable discussing, interpreting and even ordering direct-to-consumer genetic health tests. This is just one of the recent announcements from 23andMe, including news of a new tool that assesses an individual’s risk of having a severe reaction to COVID-19 if infected, and talks of going public through a special purpose acquisition company founded by Richard Branson, according to Bloomberg.
All of this is part of 23andMe’s long-term goal of expanding beyond a genealogy platform and becoming a genetic analysis and drug development platform. While regulators and privacy advocates have expressed concern over the potentially sensitive nature of such genetic information – and the sometimes unexpected results they unearth – at-home genetic tests are becoming more commonplace. We expect this adoption to continue as the public becomes more broadly comfortable with the generation of this personal data and has a greater desire for personalized medicine.
The fight against COVID-19 variants
COVID-19 vaccines are being distributed across the globe, but the emergence of more transmittable or severe variants of SARS-CoV-2 (the actual virus that causes the COVID-19 disease), are raising concern about the overall effectiveness of the approved vaccines. In an interview with Bloomberg on Tuesday, Pfizer CEO Albert Bourla had some reassuring words regarding the emerging variants: “Every time a new variant comes up we should be able to test whether or not [our vaccine] is effective,” he said. “Once we discover something that it is not as effective, we will very, very quickly be able to produce a booster dose that will be a small variation to the current vaccine.”
While the development and administration of an additional booster would further complicate vaccination efforts, it is a reassuring reminder of how robust diagnostic and drug discovery platforms have become, and gives us hope that continued development of mRNA technologies will lead to therapies for other diseases beyond COVID-19.
AstraZeneca expands portfolio with first AI-generated target
The traditional drug development process typically takes years, costs billions of dollars and often involves many dead ends. Advances in big data, artificial intelligence, machine learning and raw computing power are helping drug developers improve that process, and COVID-19 vaccines are the poster children for these new capabilities.
AstraZeneca – whose COVID-19 vaccine has already received emergency authorization from the U.K., and expects approval from the E.U. and United States in the coming weeks – has just added its first AI-generated target to its pipeline and will begin researching additional compounds based upon this platform. To be clear, this technology is still in its infancy, but by leveraging massive data sets and AI predictions, developers are able to more efficiently identify potential drug candidates and avoid dead ends.
‘Different page’ but same book on drug pricing
Last week, we highlighted several of President Joe Biden’s new appointees to key positions that will affect the life science and health care ecosystem. It is important to remember that while the administration has changed, many challenges around access to and affordability of health care and drugs remain.
An early challenge for Biden’s Department of Health and Human Services will be a series of lawsuits over the Trump administration’s efforts to lower drug prices. As this Bloomberg Law article puts it: “The Trump and Biden administrations are ‘not on the same page, but in the same book,’” when it comes to drug prices.
