Trends in Medical Research for Gene and Cell Therapies

The field of gene therapy is constantly evolving, with new innovations being developed and research being conducted all the time. The 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), May 12-15, 2021, served as a hub for showcasing these developments. One study presented by Avamab Pharma, prompted by the COVID-19 pandemic, sought to improve monoclonal antibody (mAb) therapy as a strategy for passive immunization in older adults contraindicated for mRNA vaccines by using patients as a “bioreactor” to produce neutralizing mAbs in vivo via vectored immunoprophylaxis (VIP).

Investigators evaluated the expression and safety in mice and lambs of vectorized 3 mAbs (31C2, 15A7, and CR3022) that bind the SARS-CoV-2 spike protein. The 31C2 mAb had the strongest, dose-dependent expression, with a peak average human immunoglobulin G (hIgG) plasma serum concentration of 175.1 ug/mL and an average of 276.5 ug/mL hIgG in bronchoalveolar lavage fluid in mice. No signs of toxicity were seen in mice or lambs.

GeneTherapyLive spoke with Brad Thompson, PhD, chief executive officer, Avamab Pharma, to learn more about the company’s research and how real-world events and attitudes shift the direction of funding in medical research.

Why do you think many companies haven’t investigated gene therapies for infectious diseases out of the gate?

Part of it was that the rare diseases were already trendy outside of gene therapy. So that was a place you could make money. For many years you couldn't raise money for rare diseases, getting a rare disease designation was like saying your market is small from the start.

There are some IP [intellectual property] issues too now in treating disease where the target is known and all you're doing is employing gene therapy to remedy a target. Although it can be hard to get IP, just having data exclusivity has really reduced the need to focus on IP. So, I think those are elements of why people focused on rare diseases. You can always tell in biotech where the money is because that's what people focus on.

What do you think drives trends in medical research?

In general, you couldn’t really raise money for infectious disease companies, until we have epidemics or pandemics. Just like cancer, 20 years ago—being a cancer company was a nightmare to raise money, because everybody just thought you couldn't do anything about it anyway. Advances in cancer care hadn’t been made in 25 or 30 years. The standard of care was radiation, which is still a first standard of care, and chemicals you wouldn’t want to give your children.

My former chief medical officer used to be head of worldwide Regulatory Affairs for BMS, but he started out as a radiation oncologist in the 40s. They didn't really know how to dose in cancer back then. He said they would dose until the tissue got ‘crunchy.’ But we've made advances since then in targeting and dosing radiotherapy. There used to be no money in the field, but then in the last 15 years, cancer companies have gotten access to almost every fund in the world. It’s trending, and right now you have to follow the money in biotech.

Biotech’s special skill is spending money, that's our superpower. These companies have a very different mindset. We chase money, but we don't chase sales, we chase investors. That determines what we can do. So right now, investors love gene therapy. Part of that is because of COVID, and part of that is because of presentations at big meetings like ASGCT.

Transcript edited for clarity.

REFERENCE

Rghei AD, van Lieshout LP, MCLeod B, et al. Vectorized SARS-CoV-2 human IgG Expression in mice and ovine animal models is feasible and well tolerated. Presented at: 2021 American Society of Gene & Cell Therapy Annual Meeting; May 12-15, 2021. Abstract 234.