Spotlight on Advanced Therapeutics

Advanced therapeutics: revolutionising healthcare using cell and gene therapies

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.

Working across the full spectrum of advanced therapeutic products

Immune cell therapy

We work with a biopharmaceutical company developing T cell therapies which target protein markers that can be found on the surface of cancer cells. Recent successful fundraising has enabled them to begin clinically testing their cell immunotherapy in humans. A member of our team did over 2 years on secondment focussing on IP matters related to the company's bioinformatics platform, and we continue to work on drafting, prosecution and FTO matters for them.

Regenerative therapy

We represent the technology transfer office of a leading UK university that has numerous researchers in the cell and gene therapy space. We provide advice on patentability and patent filing strategies for a wide variety of projects in this space, as well as drafting, filing and prosecuting patent applications protecting the innovations that arise and providing due diligence support for subsequent spin-out and licensing deals.

Cell Culture

We are proud to represent a UK based biopharmaceutical company that designs and delivers TCR T cell therapies for the treatment of a number of cancer indications. One of their cell therapy products is now seeking regulatory approval and other products have shown promising results in clinical trials. We work with them to draft and prosecute patent applications to protect their innovations.

AAV production

We work with one of the top research universities in America. They use gene therapy to treat a variety of genetic disease, often using AAV vector technology. Recently the institute have licensed the rights to develop and commercialise a gene therapy to treat spinal muscular atrophy to a global biopharmaceutical company. The work we do involves EP strategy and prosecution via partnerships with US attorney firms.

Gene therapy

We represent a biotechnology SME focussed on research and development of novel gene therapies to address diseases related to complement dysregulation. Our work involves strategic advice, drafting, prosecution, freedom-to-operate intelligence, and clearing third-party patent rights.

Read our Advanced Therapeutics Blogs

Efficiency in Expansion: Meeting the Scale-Up Challenge in Cell and Gene Therapies

by Katherine Collins

One of the pivotal challenges in the development and commercialisation of cell and gene therapies lies in scaling-up production. This challenge is particularly pronounced as companies transition from ...

Endometriosis - research, diagnosis and treatment progress

by Fay Allen

March is Endometriosis Action Month, and this year’s focus is on raising awareness.

Top marks for Iovance as FDA approves first-in-class cell therapy Amtagvi for solid tumours

by Katie Dale

On February 16, 2024, a real milestone was reached in the cell therapy field, as the FDA approved the first cell therapy for solid tumours. The therapy is called Amtagvi (lifileucel), a ...

Yoni Health: the medical secrets of menstrual blood

by Frances Salisbury

Menstrual effluent contains a wealth of scientific info, including biomarkers for endometriosis, diabetes, certain cancers and polycystic ovary syndrome. Yoni Health is the first start-up in this ...

Genes as Medicines: Treating the Untreatable

by Edward Couchman

In 2023, gene therapy hit the headlines in the UK, when the BBC reported the cure of a toddler with metachromatic leukodystrophy (MLD) with the gene therapy drug Libmeldy. MLD is an inheritable ...

The first CRISPR treatment – Casgevy wins UK approval for sickle cell disease

by Alex Galbraith

In a world first, the UK medicines regulator has given the green light to a therapy employing CRISPR gene editing for the treatment of disease. Remarkably, this first regulatory approval comes just ...

Businesses Developing 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.

The Reluctant Gradualist

Taking on aging

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’.