Investing in Cell and Gene Therapy: How Investors View the New Frontier of Life Science Technology

20 Jun

By Lucy Parkinson, VP of Investor Research, LSN

RESI’s panel and workshop sessions explore many topics in early stage life science funding.  At RESI Philadelphia, the Investing in Cell & Gene Therapy panel took an in-depth look at how investors approach the most complex new therapies.

The panel was moderated by Shyam Patel (Associate Director, Portfolio Development and Review, California Institute of Regenerative Medicine), and the panelists were:

  • Jane Hollingsworth (Managing Partner, Militia Hill Ventures)
  • David Xu (Chief Business Officer, Viva Biotech)
  • Peter Young (Venture Partner , Pappas Capital)
  • Hannah Shen (Associate Director, BridgeBio)

While the cell and gene therapy space is still at the cutting edge of life science technology, the field is inching closer to mass adoption.  As Shyam Patel says, following recent landmark drug approvals, there is now a huge pipeline of potentially transformative cell therapies and gene therapies in development, with a thousand clinical trials ongoing right now, and $13 billion in investment in this space last year.  “There’s a lot of money, a lot of products, and a lot of choice for both investors and companies.”

However, these therapies present unique challenges for investors.  Peter Young noted the difficulty for traditional VC investors of managing a fund with a 10-year life cycle and investing in science that takes a generation to mature to the point where it’s it’s suitable for routine use.  Additionally, the industry needs time to adapt to these new technologies, in terms of regulation, manufacturing and reimbursement.

According to David Xu, manufacturing is a key piece of the puzzle.  How can a company consistently and effectively produce a cell therapy or a gene therapy?  As an investor, he pays attention to this technical angle, along with the unmet need involved.  “If you are addressing a problem in which the unmet medical need is not obvious and you don’t have a new angle addressing the manufacturing/technical side of the problem, it’s probably not a good project to look into.”  By investing in technical improvements, it will also be possible to reduce the cost of these therapies, which at present can run to over a million dollars per treatment.

The investors also explored the regulatory issues involved, and noted that the FDA are adopting policies that enable innovation, such as being open to trials that involve fewer patients.  But there’s a challenge for startups in terms of getting the right regulatory expertise.  Per Jane Hollingsworth, “The regulatory part is difficult as there are only so many people out there who really know this space from an FDA perspective.  The FDA is overwhelmed as the result of the successes of this sector.  Now there are many clinical trials taking place and it’s incredible how busy they are.  They want to work with people who understand the pressures they’re under in this sector.  It’s important to attract the right regulatory consultants who can help your company navigate the FDA.”

Circling back to the issue of how an investor’s fund structure can make investments in new fields difficult, Hannah Shen described how her firm BridgeBio has found benefits from using a different structure, in which a subsidiary company is created for each of the assets they invest in.  Hannah says that one benefit of this model is that it makes it pososible for the firm to focus on rare disease programs as part of the portfolio.

So how do investors find the right opportunities in cell and gene therapy?  The investors’ dealsourcing methods include referrals, conferences like RESI and also scientific conferences, tech transfer outreach and academic collaborations, and of course inbound inquiries.  David said that of the pitches his team received, half didn’t pass initial screening.  As his firm (Viva Biotech) is a very early stage investor, they focus on the quality of the team and the market potential of the product.   For dealscreening at BridgeBio, Hannah Shen focuses on the scientific understanding of the asset, and whether BridgeBio’s resources are a good fit for moving the product forward.

Jane Hollingsworth went into depth on Militia Hill Ventures’ strategy – and said that issues like manufacturing and pricing are not as important in their consideration: “Either we identify a problem and look for the best way to solve it by gathering the best scientific expertise around the world, or alternatively, a really hotshot scientist comes and tells us the problems they’re working on.  It’s always the problem and the people.  If those two factors get through our screening, then manufacturing is just a detail – and reimbursement is even less so. If it’s an unsolved problem that gene therapy could solve, and the best team in the world is working on it, and scientifically it looks viable, we’re going to assume that if it’s successful it will be paid for.”

So will these new technologies become a significant piece of the biopharmaceutical market soon?  Shyam Patel has some pause.  “I would differentiate between clinically promising and commercially promising projects.  The most clinically advanced projects that CIRM [California Institute of Regenerative Medicine] has supported are focused on diseases with small patient populations, and it’s not clear if these projects will be commercially successful.  That’s where they see the most clinical success right now.  What’s commercially promising could be something completely different.”  Nevertheless, the investors continue to look for revolutionary new technologies in this space that could become success stories for their portfolios.

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