Good Day BIO: Gene therapy market will soon follow the science

We recap Day 3 of the CEO & Investor Conference with the outlook on gene therapy and gene editing, and how start-ups can get funding. (784 words, 3 minutes, 55 seconds)

Gene therapy market will soon follow the science

The science of gene editing has taken off, and the impact on health and the biotech market should soon follow, said a panel on commercialization and approval of gene therapies on Day 3 of the BIO CEO and Investor Conference.

The context: There are 900 applications for investigations on gene therapies; while FDA says they are especially effective against inherited disease, only two heritable diseases currently have approved gene therapies.

The challenges: Along with approval, challenges include getting enough experienced scientists to fill available jobs—and investment, panelists said. 

“There are going to be more wins, and when those happen, there’s going to be a different psychology in the market,” said Aruvant Sciences CEO Will Chou, who noted the market is still young. 

“For the first time in the history of our species, we’re talking about not therapies but cures, and I think people don’t quite see that point yet,” added Rocket Pharma CEO Guarav Shah. 

Another challenge is public acceptance of gene editing, said Lawrence Klein, COO of CRISPR Therapeutics: A sunburn would lead to “more DNA damage than we’ll ever produce treating a patient with one of these CRISPR-based therapies that we’ve developed, with the stringent requirements we adhere to.”

Read: Gene editing allows pig heart transplant 

Gene editing can also boost the health and sustainability of our food supply—making plants and animals more resilient in the face of disease and heat. But, just two biotech animals have been approved for food.

Read more on the live blog.

Biotech innovators need innovative funding

Brilliant biotechnology innovations can only change the world if they get into the market. Fortunately, the people financing start-ups can be just as visionary as the scientists.

Helping biotech start-ups launch—through incubators, SPACs, and mergers—was a central theme of Day 3 of the BIO CEO and Investor Conference.

At least 50 biopharmas were involved in Special Purpose Acquisition Company (SPAC) deals in 2021, and at least 50 more SPACs are currently in the market for biotech, but choosing the right fit for your company is essential, speakers agreed yesterday. 

SPACs, which raise money with the goal of investing in a company, can eliminate some of the challenges of an IPO for start-ups, said Jonas Grossman, president of Chardan Capital Markets. SPACs can be “a very elegant way” to handle an IPO while “bringing in a curated, specialist investor group,” added Michelle Doig, Partner at Omega Funds. 

What about incubators? Start-ups looking to grow into their IPO can benefit from the support of an incubator that provides lab space, professional advice, and funds. The benefits are obvious, but the challenge is to get accepted by an incubator in the first place, agreed another panel. 

NCBiotech incubator makes “a pretty deep dive and due diligence” into “team technology, IP, market competition, etc.” before selecting about 20-25 start-ups from roughly 80 applicants each year, said Vivian Doelling, NCBiotech’s VP of Investments. She advised applicants develop a “sound IP strategy.” 

Another funding trend has been M&A in the Asia-Pacific region, particularly from China, which has seen mergers in two waves: “The first wave was for the Chinese biopharma to in-license the global drugs to China markets,” said Anh Do of Indus Capital Partners. “The second wave was out-licensing of Chinese discoveries to the global markets.”

More Health Care News: 

Barron's: Biotech Is Creating the Jobs of the Future. It’s Time to Give It a Boost.
"America boasts the world’s most impressive biotech industry—by a long shot," writes BIO's President and CEO Dr. Michelle McMurry-Heath in Barron's. "The sector produces lifesaving and life-changing innovations, including two-thirds of the globe’s new medicines. And it also generates immense opportunity, directly employing roughly two million Americans and supporting over nine million jobs in total."

President Biden’s Thursday: Travelling to Lorain, Ohio, to deliver a speech on the Bipartisan Infrastructure Law, which includes $1 billion to clean up the Great Lakes. Dr. Francis Collins, who recently left his position as the head of NIH, will serve as acting White House science adviser, reports POLITICO West Wing Playbook. 

What’s Happening on Capitol Hill: The Senate is expected to act on temporary measures to avoid a looming government shutdown and extend the deadline for reaching a budget deal, POLITICO reports. The House Committee on Education and Labor’s HELP Subcommittee will hold a hearing, Exploring Pathways to Affordable Universal Health Coverage. The Natural Resources Subcommittee on Energy and Mineral Resources will hold a hearing, Securing the Future of Climate Adaptation Science at the United States Geological Survey.