It’s fair to say that Steven Dowdy, the co-founder of Solstice Biologics Inc. and an academic scientist at the University of California, San Diego, has experienced more career-related drama than normal, even for a biotech businessman.
Instead of the typical startup turmoil of fickle IPO windows and fundraising hang-ups, this scientist-turned-entrepreneur lived through being shot, allegedly by his rampaging former colleague, Hans Petersen, the ex-biotech executive at the now liquidated Traversa Therapeutics Inc.
While the attack occurred over a year ago, and Dowdy is fully recovered and working harder than ever, it made enough of a splash to taint a field of scientific research, RNAi therapeutics, which already suffered from a shaky reputation.
Now, with a groundbreaking scientific advancement to his name, Dowdy may be able to bring legitimacy and hope to a field hampered by failed research and bad press.
The researchers working in RNAi therapeutics are trying to solve a scientific problem laced with challenges — the successful delivery of RNA-related drugs into patients. Right now, less than 10 percent of the human genome is accessible when using current drug technologies. RNAi, however, offers the promise of accessing the remaining 90 percent by treating disease through genetics.
This problem has obsessed a lot of sharp minds and cost companies billions of dollars, to no avail — hence the reputation for failure. It’s a challenge that some find unbreachable, and companies in the RNAi space, such as Carlsbad-based Isis Pharmaceuticals Inc. and Regulus Therapeutics Inc. in San Diego, have sought to work around the problem rather than solve it.
So what’s hard about getting RNA-related drugs where we want them to go? There’s a whole flurry of obstacles, so get ready for some serious science talk.
If a nucleotide that carries RNA-based medicine is injected into a sick patient, the goal is for the medicine to reach its destination uninhibited. Instead, the body’s natural defenses stop it in its tracks. See, viruses have similar characteristics to RNA-based medicine, and the body has built-in security that prevents this type of molecule from circulating in the body. Assuming that the nucleotide is a foreign entity, the body commands enzymes to attack. As a result, the nucleotide is degraded and the medicine never reaches the end zone.
Unfortunately, this is just the first challenge. If by some miracle the drug arrives at the end zone, there’s another barrier. Traditional drugs are made with small molecules that can slip through cell tissue easily, but RNA drugs have big, fat molecules that can’t simply slink in. Dowdy thinks part of that problem is that the nucleotides are negatively charged, and so is the cell membrane. If you’ve ever tried to mash two magnets together with the same negative charge, then you’ll know this is indeed a problem. They repel each other.