During a warming spring week, scientists, investors, and biotech executives crammed into tents propped up outside the Washington Hilton. Every seat was filled, and a morass of people flocked outside, ignoring warnings to not block the doors and craning to get a peek at the latest advancements in gene therapy and gene editing.
Inside, the hotel was overrun with people packing every conference room, lining the hallways, and sitting cross-legged on the lobby floor to watch livestreams of packed presentations happening just feet away. There was no room at the inn.
This was the annual meeting of American Society of Gene & Cell Therapy, and its swarms were a visual and tactile representation of the mounting excitement for high-tech therapies that treat diseases at the level of the genetic code. The meeting has nearly doubled in size since 2017 to an estimated 4,500 attendees this year. But amid grumblings about the crowded quarters, most guests agreed: this is a good problem to have.
“This specific meeting has a tangibility in the air that you can cut with a knife,” said Nicole Paulk, a gene-therapy researcher at the University of California, San Francisco. “You know that when you come to this meeting you will hear something that will change your life.”
Paulk has attended ASGCT meetings every year since 2006, a time when high-profile clinical failures and patient deaths sent the gene-therapy field’s numbers and finances tumbling. Eventually, promising data from an experimental therapy led to the approval of the first gene therapy in the US in 2017, Luxturna, which partially restores sight in a form of inherited blindness. Another gene therapy, now called Zolgensma, for a childhood neurodegenerative disease called spinal muscular atrophy, is expected to earn approval this month.
Now, there are more than 100 ongoing gene-therapy clinical trials delivering corrective pieces of DNA to the brain, eye, liver, muscles and more. “It was a slow trickle that became a tidal wave,” Paulk said. “And now that we have things that look like they are curing people, there is no disease off the table.”
During a plenary session, Audentes Therapeutics shared preliminary data from a Phase I/II clinical trial using gene therapy to treat a neuromuscular disorder in children called X-linked myotubular myopathy. A striking video showed a boy with the condition who needed a ventilator to breathe and could barely move. In a video taken after the boy received the gene therapy, he was smiling and bouncing around during a doctor’s visit. For many in the audience, it was an emotional moment.
“There are not many scientific conferences where people are tearing up in the crowd,” said Emily Walsh, a cell- and gene-therapy expert at the life-science consultancy, Tremont Therapeutics.
These are the moments that many in the field live for, but turning these experimental therapies into bona fide drugs will come with growing pains. Walsh said she was pleasantly surprised to see even the meeting’s most technical presentations—covering assay development, manufacturing, and quality control—highly attended. It’s a sign of the field’s ongoing maturation.
Other jam-packed presentations highlighted methods for discovering or designing new variants of adeno-associated viruses (AAVs), which scientists use as delivery vessels for shuttling a gene-therapy’s corrective copy of DNA into cells. New AAV variants could help get more of that DNA to parts of the body that are currently hard to reach, including the brain. Presentations also offered new ways to potentially dampen the immune reactions spurred by AAV. The reactions limit its use in some people who have preexisting immunity to the virus and prevents redosing of the therapy if it doesn’t work well enough the first time.
The rush to solve such technical issues is spurred in part by new investments that signal the drug industry’s long-awaited acceptance, and financial backing, of gene therapy. Last year, Novartis purchased AveXis, the maker of Zolgensma, for $8.7 billion. And in February, Roche announced it would buy Spark Therapeutics, the maker of Luxturna, for $4.8 billion. The Alliance for Regenerative Medicine, a biotech trade organization, reported that gene and gene-modified cell therapy companies raised $9.7 billion in public and private financings in 2018, up 64% from the year before.
In fact, the traditionally academic conference’s burgeoning numbers were partly attributable to investors and businesspeople scoping out, or pitching, the latest technologies. Paulk noticed that an increasing proportion of people who approached her during poster sessions were investors. “It has fundamentally changed the nature of the meeting, in both a good and bad way,” she said. “People are hesitant to talk about things that are not published or patented, and that makes me sad.”
Denise Sabatino, who works on gene therapy for hemophilia A at the Children’s Hospital of Philadelphia, agreed that people are sharing less about their ongoing research, but she and Paulk both noted that there is a flipside. “Fifteen years ago, biotech was scared of gene therapy,” Sabatino said. Now the influx of funds means that new, experimental therapies are being moved to the clinic much faster.
That money is also creating new jobs. In the lobby, nearly 100 fliers advertising open positions at biotech companies and virus manufacturers filled every inch of two large message boards. Representatives from the Food and Drug Administration were in plain sight too, looking for new recruits to handle the surge of investigational new-drug applications for cell and gene therapies it is receiving, which doubled from 2017 to 2018.
Gene-therapy fever is also pulling some experienced drug developers out of big pharma companies. Adrian Woolfson, for example, spent more than a decade in oncology drug development and directed clinical immunooncology programs at Bristol-Myers Squibb and Pfizer. In January, he left oncology to join the gene-editing company, Sanagmo Therapeutics, as its head of R&D.
“A lot of my people were surprised that I would leave immunooncology to move into gene editing and gene therapy,” he said. “But I am good at spotting trends, not that this one is that hard to spot.”
Excitement didn’t dampen as the week buzzed on. During the last poster session of the conference, organizers turned off the lights and people griped as they were shooed away from the posters and out of the vendor showroom.
ASGCT is still small compared to other premiere scientific conferences, such as the American Chemical Society’s meeting, which numbered nearly 16,000 this spring, or the American Society of Clinical Oncology (ASCO) annual meeting, with some 40,000 attendees last summer. But some think that as the gene therapy field grows, the ASGCT conference will rival that of its scientific siblings.
“This will be like ASCO in four years,” Woolfson said. If so, they’re going to need a bigger venue.