Spotlight on Advanced Therapeutics

The human body is made up of cells which are primarily organised into tissues. Each cell contains nucleic acid, such as DNA, which contains the genetic information required for the cell to function.

Recent innovations in cell and gene therapies allow these fundamental biological building blocks to be modified and manipulated to treat disease. Gene therapy seeks to alter the expression of a gene or to modify the biological properties of living cells for therapeutic use. Cell therapy involves placing new, healthy cells into the body, for example to replace failing cells or to carry out a particular therapeutic function. Tissues can be engineered to contain beneficial cells or to replace or regenerate damaged tissue.

Important advances in gene editing, nucleic acid delivery methods (such as adeno-associated virus (AAV)), immunotherapy, chimeric antigen receptors (CARs) and T cell receptors (TCRs), CAR-expressing immune cells (such as CAR-T), cell culture technology, protein engineering, precision medicine and machine learning-approaches to drug discovery mean that halting and reversing disease, restoring damaged organs, and treating many previously-untreatable conditions is now a realistic goal.

The first wave of cell and gene therapies (also known as ‘advanced therapy medicinal products’ (ATMPs) or ‘cellular and gene therapy products’) are already being used in the clinic with striking results, and the development of next-generation technologies holds even greater promise. Such technologies are attracting significant investment: companies developing cell and gene therapies received approximately 30% (>$15 billion) of global VC biotech funding in 2019-2021.

Mewburn Ellis are delighted to be working with businesses that are developing technologies across the full spectrum of advanced therapeutic products.

A successful IP strategy must be designed and tailored to achieve the company’s commercial goals

Although companies developing cell or gene therapy products operate on a range of different business models, these models typically involve the development of therapeutic assets. In order to produce a consistent, clinical grade therapeutic product and successfully overcome the high regulatory and financial barriers required to bring it to market, a small business will typically need to collaborate or license with, or be acquired by, a larger entity, such as a big pharmaceutical company.

Even getting to this stage is likely to be an expensive endeavour. Multiple rounds of fund raising may be needed to progress an advanced therapeutic asset to a stage where it is sufficiently attractive for a larger entity to get involved. Once involved, the larger entity will then need to invest resources and money to take the asset through clinical trials into the marketplace.

All this investment relies on the parties being able to reap a sufficient reward if the asset is marketed successfully. If competitors can generate competing products as soon as an advanced therapeutic becomes successful, then the potential reward for its development is diminished: there may be no incentive for investors or larger entities to finance the asset or the business developing it. It is therefore crucial for advanced therapy businesses at all stages to ensure that their assets have the best possible IP protection to attract the investment and collaboration required for success.

Forward editor Caitlin Mackesy Davies learns how we might all be able to live long and prosper from one of the world’s foremost investors in innovation

Billionaire investor and author Jim Mellon describes his involvement in the longevity technology sector as a matter of ‘happenstance’.

As he tells me on a Monday morning Zoom call, one of his long-time partners – with whom he has set up a number of biotech companies over the past 15 years – has always been interested the question of extending the human lifespan. It was this connection that led Mellon to ‘become aware that the science of longevity was catching up with the aspiration of all of us to live healthier and possibly longer lives’.