Cell and gene therapies: Opportunities for innovation

By Dr. Deborah Phippard, Chief Scientific Officer of Precision for Medicine and Anshul Mangal, President & General Counsel, Project Farma.

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are transforming the way diseases are treated but are also aiming to cure entirely new patient populations. New applications and indications are being researched daily with the hope to address an array of rare and chronic diseases that have long been thought incurable. As researchers and developers work to transform the way we treat diseases, cell and gene therapies will continue to be the centre of innovation in the coming years.

Record breaking investments in 2021

2021 saw record-breaking investments across the board, with a 16% increase from the previous year, totalling $23.1billion raised. However, the question remains as to whether this will continue in 2022. As public markets adjust and the economy recovers in early 2022, investments in the cell and gene therapy space may plateau.

As we look to the future, many advanced therapy players are sticking to traditional cell and gene technologies like autologous cell therapies and gene therapy using adeno-associated viral vectors, but some are branching out to other models like allogeneic cell therapies, non-viral delivery methods and gene-editing technologies. Gene editing in particular is rising in prominence as the proportion of funding has increased from 38% to 45% of the total gene therapy financing over the past three years1. ViaCyte, Beam, and Verve are due to begin gene editing trials this year. Companies like Breyanzi, Abecma, and Carteyva received approvals in 2021 for CAR-T therapies while two promising tissue therapies were approved for Stratagraft and Rethymic.

A promising commercial pipeline

With record fundraising continuing to pour in recent years, the cell and gene therapy industry is experiencing exponential growth. There are currently 2,261 clinical trials taking place in advanced therapies around the world aimed at treating cancer, rare diseases, neurological disorders, diabetes, and cardiovascular diseases1.

Cell and Gene State of the Industry Briefing ARM Report Regenerative Medicine: New Paradigms January 10, 2022

The results of these trials are promising. Advanced therapies are providing new hope for patients and have the potential to revolutionise the way treatments are delivered, and possibly the healthcare landscape as a whole.

2021 saw the approval of over a dozen new products around the world with over 20 more expected in the US and Europe in the next two years including approvals from Gamida Cell, Krystal Bio, Iovance, Orchard, PTC Therapeutics, Atara Bio. Also, companies like uniQure, Freeline, Janssen, bluebirdbio, and BioMarin have two or more products in the pipeline that may be approved, and regulatory authorities estimate that there could be as many as 10-20 approvals for advanced therapies a year by 2025.

Achieving a sustainable growth model

As the industry experiences rapid growth, there are several challenges the sector must overcome to sustain this momentum and prevent bottlenecks. We are seeing a substantial talent shortage across the sector. As a relatively new industry, working knowledge of bringing advanced therapies to market is limited. In order to support the next generation of talent, cell and gene therapy companies need to put heavy emphasis on training opportunities and partnerships at the university level. As many new cell and gene products move through the pipeline, the industry must focus on innovation in research and clinical development, manufacturing automation and reimbursement.

Opportunities for innovation

Cell and Gene State of the Industry Briefing ARM Report Regenerative Medicine: New Paradigms January 10, 2022

The cell and gene therapy industry is growing rapidly around the world. In the US, the traditional innovation hubs in Boston, Raleigh and the Bay Area continue to be the leaders in the space, while new hotbeds in Maryland and Philadelphia are making industry news daily. The most notable players in the global market include China and the UK, both of which were granted approvals for several products in 2021 and have more in the pipeline in the next two years.

Companies in the advanced therapy space are currently assessing gene therapies for rare diseases as well as chronic conditions, like Verve Therapeutics’ work in the cardiac space. The possibilities for future indications are endless with opportunities to ease the organ transplant process or eliminate the need for it entirely, after a cardiac event. Companies like CRISPR, Beam Therapeutics, KSQ, and Intellia have opened the door for gene editing technologies to flourish.

Many companies are still developing autologous products but due to their personalised nature some companies are branching out. Cellevolve and Kite Pharma are just a few seeking new opportunities in the allogeneic technology space. Autologous products require customised manufacturing for each patient’s treatment, an expensive, often lengthy process. Allogeneic cell products leverage gene editing technology and a simplified manufacturing process that reduces capacity constraints and gets treatments to the patients faster. Cell and gene therapies provide exciting opportunities. As we look to the future of cell and gene therapies, innovation will drive an expansion of indications and increased access. The promise of advanced therapies provides new hope for patients around the world suffering from chronic and rare diseases that until now, have had limited treatment options. Major players in the industry are working diligently to innovate R&D, manufacturing, and policies to bring these lifesaving therapies to the patients who desperately need them.

Volume 23, Issue 2 – Spring 2022


1: Cell and Gene State of the IndustryBriefing ARM Report RegenerativeMedicine: New Paradigms January10, 2022

Author Bios 

Dr. Deborah Phippard is a leader of biomarker and drug development programmess for pharmaceutical and diagnostics companies, as well as the National Institutes of Health. She led the discovery of pharmacodynamic biomarkers and novel targets for inflammatory disease therapy and gained her PhD in Developmental Biology from the Institute of Cancer Research, London. Anshul Mangal is the President & General Counsel of Project Farma (PF) and Precision ADVANCE. Under his leadership, PF pioneered the industrialisation of advanced therapies including two commercially approved cell and gene therapies and created PF’s Advanced Therapy Manufacturing Playbook. To date, PF has led facility builds, partnered with 80+ advanced therapy organisations and 100+ life science companies.

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