This newsletter provides commercial insights into the ongoing development of the gene therapy market. The most noteworthy stories appear at the top along with our commentary.
1) Still Learning What We Don’t Know: Gene Therapy Market Impacted by Safety Concerns
Extreme market response to postulated gene therapy immunity and the unsettling results of research just made public by an industry pioneer remind us that the gene therapy market is still in its infancy. We still don’t know what we don’t know, particularly when it comes to long-term outcomes and adverse events. Inherent risk and unknowns affect both how and how quickly early therapies will be adopted and with which patients. Effective patient segmentation and communication are key and shouldn’t be underestimated even with successful clinical results.
Two unrelated reports emerged this month questioning the safety of gene therapy treatments, one of which sent Intellia, Editas, and CRISPR stocks tumbling $500 million (temporarily). This extreme reaction to the news that many people could be immune to CRISPR-Cas9 technology demonstrates the immaturity of the gene therapy market and how much we still don’t know.
Another report, authored by Dr. James Wilson, revealed that nerve and liver damage occurred in animals who received gene therapy treatment for SMA. Humans have responded well to gene therapy for SMA but Dr. Wilson’s caution to carefully monitor subjects underscores the fact that we still don’t know the long-term effects of gene therapy treatment or the potential result of overdosing.
One possible solution to safety concerns comes from an unexpected source: Microsoft. The company has developed a new tool, Elevation, that uses artificial intelligence to predict off-target effects. This tool may identify patients who would respond optimally to certain gene therapies and track patients and treatment reactions over time.
- BioRxiv: Identification of Pre-Existing Adaptive Immunity to Cas9 Proteins in Humans
- Human Gene Therapy: Severe toxicity in nonhuman primates and piglets following high‐dose intravenous administration of an AAV vector expressing human
- Nature: Prediction of off-target activities for the end-to-end design of CRISPR guide RNAs
- Genetic Engineering and Biotech News: FDA Imposes Clinical Hold on Bellicum Lead Candidate BPX-501
2) Headline “Gene Therapy Could Make Cancer Care More Unequal” Probably Not True
A recent article in MIT Technology Review used that headline to grab attention, but we maintain that gene therapy is not more unequal than other types of treatments for rare diseases, especially when other therapies cost $475,000 and many patients already travel to leading cancer centers for treatment. Access to care in the US is a system problem, not just a gene therapy problem.
Due to the cost and required expertise for effective treatment, most companies commercializing gene therapies will rely on a “centers of excellence” distribution model. An interactive map in the article demonstrates the treatment sites for Kymriah and Yescarta.