Currents of Disruption: Gene Therapy Offers Rare Hope
Our understanding of the genetic causes of disease has reached a critical stage, putting us on the cusp of being able to develop functional cures for a large number of rare genetic disorders, says Global Life Sciences Co-Portfolio Manager Andy Acker.
- Gene therapy has the potential to provide “one and done” treatments that can correct genetic defects that cause life-threatening diseases.
- Biopharmaceutical companies are working on treatments for the 7,000 or so known genetic disorders, including hemophilia, muscular dystrophy and spinal muscular atrophy – the biggest genetic cause of infant death.
- As investors, we recognize the importance of looking not only at large, public companies working on gene therapies, but also at the smaller innovators that have the potential to be disruptive.
Andy Acker: So when we think about gene therapy, we’re thinking about replacing a defective or missing protein. And essentially the way it works is a gene can be inserted into a carrier, for example, a viral capsid, that can then be injected into a patient and will infect the cells and actually turn them into a bio-factory that will start producing that missing or defective protein. That would be in contrast now … today scientists are also working on gene editing, where we can make direct changes to the genome, for example. Or cellular therapies, which like a CAR-T, which is a new way of taking cells out of a patient, reprogramming them to attack or kill a cancer cell, and then reinjecting them into the patient. These are all new and exciting ways of treating patients today.
Ten to 15 years ago, scientists were working on gene therapies, but they were largely ineffective. What’s happened in the subsequent years is now the techniques have gotten to the point where now we can produce 100,000-fold higher levels of a missing protein than we could 10 or 15 years ago. And that has made all the difference. So now just recently in late 2017, the first gene therapy has been approved in the United States. This drug, LUXTURNATM, is for children who would have gone blind without this treatment. So it treats a specific form of a cause of blindness. But now scientists are going even beyond that, and they’re developing new treatments for diseases like spinal muscular atrophy, the leading genetic cause of infant death, and also new treatments for hemophilia and ultimately muscular dystrophy as well.
So these are just a few examples of the more than 7,000 genetically identified diseases, and today fewer than 5% of those actually have treatments. So this will be a really important area for scientific achievement and for patient outcomes in the coming years.
So spinal muscular atrophy is the leading genetic cause of infant death. It affects about 20,000 people worldwide. And patients that are the most severely afflicted will never even be able to sit upright, and most of them won’t live to their second birthday. Now there are new treatments, some recently approved, that can improve the quality of life for these patients. But even more exciting is a potential curative approach, which is a gene therapy that actually replaces the missing protein for these patients. And the first roughly dozen or so patients that have been treated, all of them have now lived beyond two years and many of them are starting to stand and even walk.
These are advances not only for patients, but for investors as well. The company that developed this new gene therapy was recently acquired by Novartis for almost $9 billion.
So these are really game-changing types of advancements that can have a huge impact on patients and improve their quality of life and ultimately their survival and potentially also create very attractive opportunities for investors.
When looking at these disruptive companies, it’s important to understand that not all of them are public companies. And so we believe it’s important to also look at the earlier-stage companies that we think can be disruptive in terms of addressing unmet medical needs.
The health care industries are subject to government regulation and reimbursement rates, as well as government approval of products and services, which could have a significant effect on price and availability, and can be significantly affected by rapid obsolescence and patent expirations.