The Alliance for Regenerative Medicine’s (ARM’s) annual Cell and Gene Meeting needs a bigger boat. Now in its 13th year, the conference attracted more than 1,200 people to Torrey Pines Mesa in San Diego, from October 3 to 5. Last year it drew around 900.
“It’s rare that you have pretty much everybody in your subsector of biotechnology in one place at one time,” said Eric Soller, vice president of Corporate Development and Strategy at BlueRock Therapeutics.
This was a far cry from the first meeting in 2006, when around 350 scientists huddled for warmth against a hostile world. Early gene therapies had ended in disaster, with the deaths of Jesse Gelsinger and others, and federal restrictions were hampering stem cells. No one knew if any of these approaches would succeed and a betting person might have taken the short.
Not anymore. Recent successes, such as CAR-T therapies and Luxturna, have shown that cell and gene therapies offer more than just potential. Companies are trying to catch that wave, and the meeting is as much about deal making as it is about science.
“I’m not able to go to the presentations because I’m having meetings with existing partners,” said Soller.
BlueRock is leveraging pluripotent stem cells to engineer therapies for autoimmune conditions, cardiology and neurology and will soon begin clinical trials to restore dopaminergic neurons in Parkinson’s patients. But the company believes their platform could be useful in multiple indications, far more than they can afford to develop on their own – which is why Soller had such a busy conference. It’s a target rich environment.
“All major pharma companies are active in this space,” said Gil Van Bokkelen, chairman and CEO at Athersys in a phone interview. “They’re either looking for opportunities or they have their own internal programs.”
This quest for new partners is central to the meeting. Organovo, one of only two San Diego companies to present this year, is in the middle of a business pivot. The company initially focused on selling their 3D-printed liver and kidney tissue to drug companies for preclinical toxicity studies. They still do that, but have largely segued into liver disease therapies.
“As we understood the tissue more in the lab setting, we became more positive about the ability to use the same tissues at higher scale to implant in humans to replace missing functionality,” said Organovo president and CEO Taylor Crouch in a phone interview. “We’ve been shifting our priorities, so now our number one focus is the therapeutic track.”
Last year, the company received FDA Orphan Designation to treat Alpha-1 Antitrypsin Deficiency. Crouch believes a printed liver tissue patch could supply the missing enzyme and mitigate symptoms.
“You can imagine all kinds of combination of technologies and strategies,” said Crouch, “like-minded companies exploring synergies and ways to collaborate.”
The sector has grown up around difficult projects: orphan indications and conditions that drugs and devices can control but never solve. Athersys is applying their stem cell product, called MultiStem, to help patients with heart disease, inflammatory and immune disorders and particularly stroke.
“We can administer MultiStem as a true off-the-shelf allogeneic cell therapy product without having to tissue match it and without having to immune suppress the patients we’re treating,” said Van Bokkelen. The company just launched a Phase III trial for ischemic stroke in the US and Japan in collaboration with Healios.
The MultiStem stroke product has received Fast Track, Regenerative Medicine Advanced Therapy (RMAT) and additional designations from the FDA and other agencies that could accelerate approval. Van Bokkelen chaired ARM in 2011 and points to these regulator refinements as another way the sector has pushed forward.
“ARM was built on the idea that we’re going to work together to solve some thorny issues,” he said, “such as working with the FDA and others to clarify the regulatory standards.”
The meeting’s first two days were consumed with business, but on the last day the action moved over to the Salk Institute, where companies and academic scientists presented their research.
A presentation from Brian Kaspar, chief scientific officer at AveXis, now part of Novartis, validated how far the sector has come. The company has developed AVXS-101, a gene therapy for spinal muscular atrophy (SMA). Kaspar showed heartbreaking videos of SMA infants with virtually no mobility. Only 8 percent of these babies make it to 20 months.
In the Phase I trial, 15 children received a single dose, and the results have been spectacular. To press the point, Kaspar showed video of a patient challenging her doctor to a pushup contest. AveXis has begun dosing patients in a pivotal Phase III. It summarizes the conference’s overall message: gene and cell therapies have arrived.
Photo: jxfzsy, Getty Images
