A year after a Chinese scientist unleashed a storm of condemnation by announcing the birth of a baby whose genes he had edited, the medical and scientific community has yet to agree on rules guiding research into the practice.
Still, that hasn’t dimmed venture capitalists enthusiasm for the technology, with its potential to create medical treatments and huge profits.
The five leading privately held companies in the space have raised more than $1 billion this year, up 72% over the roughly $600 million they netted from funding rounds in 2018, according to Genetic Engineering & Biotechnology News. The total includes Poseida Therapeutics’ $142 million Series Cround in April and Beam Therapeutics’ $135 million B round a month earlier.
Chinese scientist He Jiankui used CRISPR technology to create HIV-resistant embryos but earned widespread condemnation from the scientific community who raised safety questions. Earlier this year Russian biologist Denis Rebrikovwas condemned for his announced intention to follow Jiankui and edit an embryo’s genes.
In the aftermath, scientists have engaged in a fierce debate about the need for regulation. In a letter in the publication Nature this year, a leading group of ethicists and researchers argued for a ban on gene editing, but others believe that self-regulation will remove obstacles without creating undue risk.
Big pharma companies remain optimistic about gene editing. In August, Bayer agreed to pay up to $600 million to acquire BlueRock Therapeutics. BlueRock’s stem cell therapies help combat neurology, cardiology, and immunology disorders.
In June, Vertex Pharmaceuticals acquired Exonics Therapeutics as part of a more than $2 billion expansion in the space. The company will also spend more than $1 billion on a continuing partnership with CRISPR Therapeutics that has resulted in the first gene therapy to be considered for approval by U.S. regulators. CRISPR technology enables companies to change DNA sequences and alter gene function, and its stock has more than doubled this year.
Governments have been wrestling with the issue of gene regulation. In May, China introduced a regulation that would hold scientists involved in gene-editing responsible for the outcome. About 30 countries have passed gene-technology legislation.
Meanwhile, the World Health Organization has proposed creating a registry for tracking gene-editing clinical trials. But the registry will be optional and critics of the WHO plan say it does not improve on current monitoring programs, which include the U.S.-based ClinicalTrials.gov.
- Publicly traded Sangamo Therapeutics and Horizon Discovery Group generated $85 million and $71 million, respectively, in revenue last year.
- A Global Market Insights report predicts that the gene editing industry will generate $2.5 billion in revenue by 2025.