If anyone needs evidence of the vast gulf that exists between the hype and the reality of gene therapy, he can find it in the contrasting fortunes of Neurogene and Sangamo, two companies at the forefront of this sector.
The former, established just a year ago, has just closed a Series A Bond sale worth nearly $70 billion to support the therapies it is creating for neurological diseases. The latter, which has been doing its own research for 20 years, has just lost a third of its stock market value — because it still can’t find a winning formula.
Progress is certainly being made in gene therapy. Sangamo’s latest clinical trials may have prompted the brutal sell-off on Wall Street. But they also suggest that the company has broken new ground by achieving “in-body” gene-editing, news that would cheer researchers everywhere.
The FDA, which recently approved a string of gene therapy treatments, is certainly on board. It says we are approaching a “turning point” and predicts that it will soon be approving twenty new therapies a year.
At the moment, the facts tell a different story. The promise of gene therapy — a single-shot panacea, one that’s quick enough to catch childhood diseases with far fewer complications than traditional drug-based courses — is yet to be matched by actual advances in the laboratory. While it’s easy to see why regulators, and venture capitalists, are so enthused, that enthusiasm could be dangerous unless kept in check.
Concerns about the viability of gene therapy have, until now, largely focused on safety. Yet an even bigger worry is the apparent ineffectiveness of many experimental therapies. While many of the latest trials report strong gains on safety, with patients reporting few if any adverse effects, they also show precious little evidence of positive change. read more