One part of the biopharma space, an area I specialise in, is cell and gene therapy. This involves either the alteration or transfer of genetic or cellular material into an organism to better help the host fight or deter some sort of medical condition.
To put it super-simply, cell and gene therapy involves going into the most fundamental building blocks of a person and altering them in some way to make that person work better.
The State of Play
According to the Alliance for Regenerative Medicine, a body which monitors the wider biotechnology space, for such a new technology the market is in a period of explosive growth. Whilst there are very few FDA-approved technologies to date, there are over 900 regenerative medicine companies around the world today, all of which are chasing the ‘holy grail’ of FDA approval.
58% of the treatments in development today are focused on oncology, but trials are also ongoing to develop treatments to prevent a range of other conditions, ranging from geriatric diseases to dermatological conditions to congestive heart failure.
The companies involved are receiving major backing, from governments, private equity and other sources, with $13.3B raised in 2018 across the whole market. That's an increase of 73% on 2017 figures.
2018 saw many companies going public like Allogene in October, and some huge partnerships were made, between companies like Spark Therapeutics, who signed a $110M up front agreement with Jazz Pharmaceuticals and a separate agreement worth $105M with Novartis. 2019 has carried on where 2018 left off, as we've already seen Biogen purchasing Nightstar Therapeutics in a deal worth $800M.
Simply put, cell and gene therapy is one of the most exciting markets in the world, despite there being relatively few treatments on the market. But who has received approvals to work in front line medicine?
Whilst 2018 saw over 1000 clinical trials take place, involving thousands of patients, very few have received FDA approval and are available for public use in the USA – the aspiration for all medical treatments currently undergoing development.
Two such treatments are Kymriah from Novartis and Yescarta from Kite Pharma, which both utilise CAR-T therapy. This involves modifying the white blood cells of the patient, effectively training them to attack cancerous cells in the body. Each targets a different type of cancer and is available to patients for whom at least 2 other treatment types have failed.
The treatments themselves are extremely complex to give out and have to be tailored to each patient individually at significant cost. The Novartis treatment is being offered to patients on the NHS in the UK, for example, but will cost approximately £282,000 per patient to administer.
As well as the massive costs attached, there have also been other issues, and even fatalities, during clinical trials when the T Cells within the patient have turned on the patient, attacking more than just the cancer cells.
There have also been notable successes as far back as 2012, when six-year old Emily Whitehead was seriously ill with leukaemia, having relapsed twice, after unsuccessful chemotherapy treatment. Still in clinical trial phase at the time, she was enrolled and managed to make a full recovery. You can read more about that story here.
Solving Bigger Problems
Cell and gene therapy also has massive potential to make big changes in other areas of our lives, in addition to improving human health. For example, gene editing could make our crops more nutritious, disease resistance or able to grow in more difficult conditions.
As the global population continues to rise, this application could have a massive impact on millions of lives. Similarly, the technology could be used on animals to help them become more disease resistant, eliminating devastating outbreaks of conditions like foot and mouth disease.
Future of Gene & Cell Therapy
In addition to those with FDA approval in the US, there are around a dozen types of therapy on the market in Europe. But with the amount of trials that took place last year it’s safe to assume that we’re going to see many more arriving on the market in the next few years.
This is further compounded by the amount of continued investment in the area. Novartis recently announced they were investing over €80M into a new Swiss facility, set to open in 2020 and other major pharma and healthcare companies are following suit.
Such is the revolutionary potential of this technology it’s impossible to estimate how far it can go and exactly what it can mean for the future of human healthcare. At the current high price point and complexity of the treatment, the next challenges are likely to come from commercialisation and payment models.
However, even taking those into account, it’s safe to say that cell and gene therapy definitely is going to change the world.
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