Biotech – The Cutting Edge: small potatoes, peak C&GT & sangreal

This week we see some more signs that the UK Government is starting to ‘walk-the-walk’ with investment in UK Life Sciences. There are sweeping policy changes that we believe need to be made to see material capital inflows to the Life Sciences Industry. It is small potatoes for now, but at least it is a start. In addition, we highlight the impending step-up in cell and gene therapy approvals (Vyjuvek, new and approved this week) and how ‘naked mole rats’ might be the answer to 21st Century beauty treatments.

A drop in the ocean or the ocean in a drop? We are outspoken about how the government needs to do more to support UK Life Sciences (PH’s UK Budget Note), budget initiatives such as an enhanced R&D tax credit and planned reform of MHRA to expedite approvals is a step in the right direction. Now, the government are to unveil a package of measures to further boost the sector. This is expected to include £150m of funding to the UK Biobank to expand research capacity (eg a new facility in Manchester) and a £38m fund to incentivise investment in Biomanufacturing. While this is a start, there is much still to do to encourage capital inflows. This includes changing restrictions as to how pension funds can back illiquid/high-growth assets, broader tax-breaks for investment in high-growth sectors and fostering closer ties with Pharma. For example, AstraZeneca’s CEO has previously criticised having to pay for the “explosion of NHS costs” as the tax on NHS drug sales have risen sharply. We also note The Times today described how Jeremy Hunt is exploring ways to encourage Pharma to run trials here in the UK. Perhaps a ‘Peel Hunt thought-piece’ is on the way…

When is ‘peak’ gene therapy? If you thought 2022 was a big year for cell & gene therapy (C&GT) approvals, think again. It seems 2023 will be bigger, with up to 13 cell/gene therapy approvals in the pipeline (making FDA’s prediction of 10-20 C&GT by 2025 a possibility). This week, the FDA adds another to its approved list: Vyjuvek for dystrophic epidermolysis bullosa (a rare disease with no current treatments) where skin becomes ‘paper-thin’ due to a lack of collagen with sufferers called ‘butterfly children’. The therapy is expected to cost c.US$631k per patient, per year, and requires chronic topical application. However, the results are clear; the patients’ skin is shown to heal faster following treatment, with patients able to take baths and sleep without experiencing severe pain.

To be (old/ratty), or not to be? It is a mission of ours to debunk myth wherever we find it, but once again, it appears like there might be more to the ‘snake oil’ of hyaluronic acid in skin treatments. Mice in the lab, given a gene from a 'naked mole rat' have shown increased production of hyaluronic acid, which is long proposed to have a rejuvenating effect on skin, and often used in anti-ageing products. These gene edited mice were found to live 4% longer and also be less likely to die from cancer (57% gene edited mice deaths vs 70% of the control mice). Whilst the mechanism is not fully understood, it is thought to retain water in tissues and exhibit anti-inflammatory properties. Although this is fascinating, we are not sure that anyone looking for anti-wrinkle cream might want a naked mole rat anywhere near them…