From promise to reality: Unlocking the true business potential of genomic medicine

The genomic medicine space has seen incredible success, but investors are overlooking it. For once, it’s not efficacy that’s the problem: it’s turning the modality into a viable business case.

In this opinion piece, Stephane Boissel, CEO of genomic medicines company SparingVision, calls for the need for three elements to solve this conundrum and allow the space to progress to the next level: (1) greater investor support of the industry to tackle more prevalent diseases, the next paradigm shift; (2) focus on lowering the costs of manufacturing and development for less prevalent diseases; and (3) build a critical mass of assets and technologies in a dedicated therapeutic area.

2023 was a watershed year for genomic medicine, marked by five groundbreaking approvals, including Casgevy, the first-ever CRISPR therapy. This trend is expected to continue, with the FDA estimating between 10 and 20 gene therapy approvals annually from 2025. By mid-2024, the FDA had already approved a total of 36 gene therapies to date, and up to 8 more approvals are anticipated by the end of the year, indicating a strong momentum in the field.

Gene therapy’s promise is unimaginable for many other drug modalities: making a deaf baby hear again, preventing the progression of blinding eye disease, curing sickle cell disease. This game-changing science is also supported by a very receptive FDA, with Peter Marks, director of the Center for Biologics Evaluation and Research, championing supportive measures of the sector across a multitude of areas to accelerate approvals. For a sector that barely existed until a decade ago, from an efficacy and regulatory standpoint, the future looks bright.

But, there is one big fly in the ointment. The sector just can’t seem to demonstrate the essential formula for return on investment (ROI). High development and manufacturing costs, combined with small patient populations and one-and-done treatment regimens, mean that, despite the incredible feats these therapies can achieve, the realities of delivering them in health systems that are designed for extended sick care, rather than cure, is incredibly difficult. To ensure the sector can rapidly move to deliver the true promise of genomic medicines, three areas need focus:

1. Greater investor support to tackle more prevalent diseases, the new paradigm following the first era focused on rare, monogenic disorders

For the ROI formula to succeed, we need to be treating much larger patient populations and pushing the modality into more complex diseases. The science to do this is there, this isn’t something we are waiting for. At SparingVision, for example, we are looking at Retinitis Pigmentosa (RP), where the global patient population is 2 million, but we also have begun evaluating our lead product in the treatment of Geographic Atrophy, which is an even larger opportunity of 10 million people. With another product we are also looking at the potential in glaucoma, which has a prevalence of more than 76 million people globally.

There are a multitude of other approaches and other companies who have the potential to do this as well, but they are struggling to attract investment. Investors of course need to be highly selective on the approaches they back, but they need to make sure that their investments focus on these high value indications, whilst having the patience for these opportunities to play out. Once we can demonstrate the ROI in larger patient populations, the nut will be well and truly cracked and the value will pour out of genomic medicines.

2. Focus on lowering the costs of manufacturing and development for products addressing less prevalent diseases

The industry cannot neglect the rare diseases space. It is our duty to find solutions, and it is painful to see promising development being stopped due to lack of investor support in the absence of future ROI. This requires players in the biotech sector – from companies to investors and regulators – to tackle several key challenges.

First, we should adopt thinking similar to the FDA’s Bespoke Therapies strategy: leveraging the development of one product to more efficiently and rapidly develop other products with a similar technology backbone. For example, we can explore opportunities where technologies become plug- and-play, such as developing CRISPR therapies that can be easily customised for various genetic conditions.

Secondly, creating standardised manufacturing protocols to ensure consistent and efficient production processes across different therapies. Finally, enhancing collaboration with CDMOs to increase production capacity and reduce costs. Coupled with the supportive regulatory environment championed by the FDA, these steps can truly unlock the potential of gene therapy for larger populations, making these revolutionary treatments more accessible and financially viable.

3. Build a critical mass of assets and technologies in a dedicated therapeutic area

Let’s face it, a small company looking to address a tiny, rare patient population with one genomic technology might get some early VC support, but it’s not going to be able to stay the course and build itself into an enduring biotech company. And a medium size company trying to deploy multiple genomic technologies across several therapeutic areas will get funded when times are good, but will face immediate existential danger when times are rougher.

To optimally advance genomic medicine at the biotech level, developers need to have scale and build a critical mass of assets, while focusing on synergistic technologies and a limited number of indications within the same therapeutic space. This approach not only allows for focus on the high value assets, but also enables the concept of “Scientific Spillover”, a term coined by Rodino-Klapac and colleagues to describe how knowledge gained from the development and production of gene therapy extends beyond the therapy and its indication, helping accelerate further gene therapy enhancements and expediting design and manufacturing fixes. At SparingVision, for instance, this is a core part of our strategy: to have a technology-agnostic approach and cross-fertilise learnings from each element of our technologies, from AAV gene therapy to CRISPR-Cas9, while focusing exclusively on the ocular space.

In summary, we all know the magic formula to success in biotech: efficacy + clear improvement on standard of care + a robust market willing to pay for it. For genomic medicines, we need to sort this final element,but investors pay attention: the sector is busily working on these challenges, you just need to make sure you are onboard with the right companies when they solve them.