PAF: Gene Drive Report

By Mack Darrow, Eric Gastfriend, John Min, and Alex Sakatos.

This is the Executive Summary of the final report from a Philanthropy Advisory Fellowship project on Gene Drive research. The full report is available here. The companion spreadsheet is available here. This research was conducted on behalf of PAF client Thomas Mather.

Gene Drive Research Funding Recommendation Report

Executive Summary

The purpose of this report is to provide recommendations to philanthropists interested in funding a new genetic technology called “CRISPR gene drive.” This synthetic biology technology allows scientists to design genetically modified animals that can rapidly spread particular genes through wild populations. As a tactic against parasitic diseases, it could be used directly on parasites to crash their population (e.g. by forcing them to only have male offspring, who in turn only have male offspring), or against intermediate non­human hosts (such as mosquitos) which grant the host immunity to the parasite, thus interrupting the parasite’s life cycle. Although the initial cost to research and develop gene drive systems are high; once developed, it offers an incredibly cost­effective means of combating infectious diseases as the gene drive is capable of spreading itself with little additional cost or human intervention. This is in stark contrast to additional anti­parasite public health programs campaigns who depend on costly massive drug manufacturing and distribution campaigns. Although many questions about this technology remain unanswered, we are optimistic about the potential of gene drives in strengthening the public health arsenal and reducing worldwide human suffering. 

In this report, we present data from various sources on the top 20 transmitted diseases and parasites with the heaviest health impacts in the world. We analyzed this data to determine the top afflictions suitable for targeting via gene drives. We used the following selection criteria:

• Global disease burden 
• Current funding environment 
• Genetic tractability
• Targeting efficiency 

This full analysis is presented in the companion spreadsheet to this report. Based on the analysis, we chose to recommend the following diseases for further research funding: 

• Soil­Transmitted Helminths (STH)
  • Hookworm
  • Whipworm 
  • Threadworm
• Aedes Mosquito borne diseases 
  • Dengue 
  • Chikungunya
  • Zika 
• Chagas disease

Recommended labs to fund: 

• STH 
  • Dr. Matthew Berriman, Sanger Institute 
  • Dr. James Lok, University of Pennsylvania 
• Aedes aegypti  
  • Dr. Omar Akbari, University of California, Riverside 
• Chagas Disease 
  • Dr. Najib M. El­Sayed, University of Maryland 
• Basic research and safety 
  • Dr. Kevin Esvelt, MIT Media Lab 

Our analysis, recommendations, and rationales for these diseases are contained in this report. However, it is important to note what we have not included in our scope. We have not considered the political barriers to implementing gene drives, which may be greater than the scientific ones. Much research remains to be done in the area of gene drive safety and control, which we recommend funding. Even with high levels of safety, however, public perception and political feasibility remains a concern. Such a modification of the environment and biosphere is unprecedented in human history, although the case of GMO crops provides some lessons. How will states react when a gene drive, introduced by a neighboring country, spreads into their sovereign territory without their permission? Such questions still need to be addressed, but investing in public education would likely ameliorate these issues. 

Although still in its infancy, gene drive research has the potential to make enormous positive impacts on global human health, and funding to answer basic questions and develop initial proofs­ of ­concept may lead to increased academic and donor interest for this transformative emerging biotechnology.

The full report is available here. The companion spreadsheet is available here.