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Gene Therapy: Sensational Outcomes. 6-figure Costs.

If you look-up almost any article on gene therapy, two things stand out. The first is just how recently gene therapy has come to market and the second is how jaw-droppingly expensive the treatments are. The cost of treatment so seems to stun journalists that the price tag itself becomes part of the story.

Case in point: last August, in a story about the first ever FDA-approved gene therapy treatment (for Leukemia), the New York Times included the $475,000 price tag in its headline. In October, Reuters repeated the convention in their story about a $373,000 Lymphoma treatment. In January, the Washington Post followed suit reporting on an $850,000 treatment for retinal dystrophy.

You cannot, it seems, talk about gene therapy without talking about costs.

In fact, gene therapy’s price tag seems to be the fastest way into many of these stories with articles openly considering whether one-million-dollar miracle treatments may or may not be outrageous. Goldman Sachs analysts have even wondered in a released report whether curing patients is a sustainable business model. — They concluded it wasn’t.

Despite the fact that these new therapies are expensive, if they work as expected, they’ll likely save the healthcare system money over time in reduced hospitalizations, deaths, etc.

For insurers charged with evaluating whether to fund such treatments, the issue isn’t whether they are too expensive but whether they are permanent. See, in order for a drug to come to market, it has to demonstrate efficacy for a certain amount of time — though not necessarily forever. Even as experts see gene therapy as a yellow brick road of cures, insurers counter that the only mechanism to truly establish whether something is a cure is time.

Nonetheless, the medical technology seems to finally be having its moment. Thanks in part to the recent discovery of CRISPR and advances in artificial intelligence, gene therapy may forever alter the way humans treat and prevent disease.

So far, a variety of explorations into gene therapy have yielded promising results. The University of Utah has curated a great list of gene therapy successes. They report several promising therapies for diseases that include:

  1. Severe Combined Immune Deficiency (SCID)
  2. Adenosine deaminase (ADA) deficiency
  3. Hereditary blindness
  4. Hemophilia
  5. Sickle Cell Disease
  6. Fat Metabolism disorder
  7. Cancer
  8. Parkinson’s Disease

Gene Therapy

According to Achars, gene therapy is “a novel approach to treat, cure, or ultimately prevent disease by changing the expression of a person’s genes.” Genetic disorders are typically the result of proteins not being made correctly — or not being made at all. Gene therapy works by deactivating, repairing, or replacing dysfunctional genes so that they, well, function. Much like a programmer might alter a few lines of buggy computer code, scientists hope to one day be able to go into our DNA and tweak it, sequence by sequence.

For this to happen, however, researchers must (a) develop methods for delivering gene therapies and (b) deepen their understanding of our genetic code. Right now, researchers are particularly interested in how genes relate to disease expression. For diseases believed to be caused by a single gene, this process is fairly straightforward. Already, scientists have identified some 10,000 diseases that fall into this category.

The difficulty, however, lies in pinning down diseases that are caused by multiple genes or multiple gene expressions. It’s far easier to find an out-of-tune piano by playing each key. For diseases caused by multiple genes, the genetic song becomes much more complex.

Thankfully, artificial intelligence may save the day.

2 1

As we’ve previously mentioned, one of the things that artificial intelligence is great at is identifying patterns. Computers have an exponential ability to mine billions of data points and draw conclusions. In this way, artificial intelligence is a bit like Neo who after years of seeing the world, finally sees the Matrix. (No spoilers but Neo is like AI for other reasons too.)

When it comes to cracking the genetic code, artificial intelligence can profoundly accelerate efforts.

Case in point: ATUM, one of the first US companies to enfold artificial intelligence into their biotech offerings, recently leveraged AI to make frog transposon 300 times more efficient at introducing genes into mammalian cells. As the article explains, a transposon is a “DNA sequence that can change its position within a genome, creating or reversing mutations and altering the cell’s genetic identity.” In other words, AI helped ATUM refine its methods for altering genetic code using frog transposon as a delivery mechanism.

Another example: one of the most groundbreaking innovations in genomic medicine is CRISPR-Cas9, a gene editing tool which Radiolab recently described as a “ninja-assassin-meets-DNA-editing tool”.

This year, several studies will kick off in the US and in Europe to investigate the power of this tool to modify our genes. However, an incorrect genetic snip here or there can lead to adverse—and in some cases, unforeseen—side effects. To improve the accuracy of CRISPR editing, Microsoft has recently released a free tool called Elevation that uses AI and genomic data to help scientists anticipate and avoid such negative outcomes.

Gene Therapy 2

For our part, Propel(x) has worked with several gene therapy companies to help them raise necessary capital for further development of their biotechnology. To date, these companies have raised over $6 million on our platform.

San Francisco-based startup Ligandal is among them. The company is working on the delivery conundrum mentioned earlier: how to get gene therapies to go to the parts of the cell that most require it. They are currently developing nanotechnologies that sends CRISPR-like interventions “exactly where they need to go.” Speaking at the Cavendish Health Impact Forum in February, founder and CEO Andre Watson asked the audience of health and science leaders, “What good would Amazon be without shipping?”

Another Propel(x) company, Haystack Sciences, is developing a series of biotechnologies that leverage synthetic chemistry, Darwinian evolution, and machine learning to facilitate and accelerate the drug discovery process. By making it easier for labs to identify new pharmaceuticals, Haystack Sciences hopes to streamline the drug-discovery process and save money. Also based in San Francisco, the company is currently in stealth-mode with their super-secret one-page website.

Screen Shot 2018 04 16 at 4.52.14 PM

4In a recent analysis, Market Reports Center (MRC) surfaced many data points, which highlight the exciting opportunity represented by gene therapy. Presently, there are 2,200 related clinical trials underway, with 55% of them taking place in the US. Additionally, the FDA recently, officially fast-tracked its approval process for gene therapies, which means that more treatments should soon come to market.

MRC lists Spark Therapeutics, Avalanche Biotech, uniQure, Voyager Therapeutics, Editas Medicine and GenSight among the many biotech companies that are raising money in this space. In the last five years, venture capitalists have invested $600 million.

As research in this space continues to accelerate with many, many different treatments rolling off of the medical production line, the biggest winners from a gene therapy revolution will, of course, be the patients who may enjoy a healthier, longer life.

 


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