Cell and gene therapy continues to provide a better standard of living for millions of people with serious health conditions, by altering or transferring genetic or cellular material to help fight and deter a multitude of medical conditions.
The technology represents a major leap in personalised medicine, enhancing our ability to treat and potentially even cure many intractable illnesses. Each cell and gene therapy is designed based on detailed information about the roots of a patient’s disease maximising the effectiveness.
Many companies innovate within this impressive space, in this article I explore five start-ups doing important work that you need to know about.
Neogene Therapeutics is a pre-clinical stage biotechnology company aiming to change the paradigm of treatment for solid cancers. Founded in 2018 and based in Amsterdam, Neogene Therapeutics provides cancer patients with T cell therapies targeting mutated proteins called neo-antigens, which are present in cancer cells because of DNA mutations.
Carsten Linnemann, the CEO, aims to develop novel technologies to enable the engineering of ‘designer T cells’ that display an optimised ability to seek and destroy cancer cells.
The company’s potential was realised when the Amsterdam-based MedTech start-up raised €93 million only two years after its inception. Neogene’s valuation is currently estimated at around half a billion, and both the US and Amsterdam teams are growing rapidly.
Mnemo Therapeutics is another biotechnology company focused on developing powerful T cell therapies that create accessible cures for solid tumours and blood cancers.
Founded in 2019 and based in France, Mnemo Therapeutics’ EnfiniT platform (a new ground-breaking drug discovery engine) leverages a new class of antigens with greater tumour specificity and a suite of technologies to significantly improve T cell memory, persistence and sensitivity. The result of the treatment is a dramatic improvement in the body’s immune response to overcome disease. Menomo Therapeutics path is created by decades of breakthrough research from Institute Curie and Memorial Sloan Kettering.
CEO Alain Maiore’s mission is to create the most powerful immune therapies to deliver accessible cures to all patients in need. The Paris based company set the record with France's largest series A funding round, raising $90 million of capital. The funding will enable the company to accelerate the development of the T cell therapy platform bringing the treatment to the market considerably quicker than first expected.
Cabaletta Bio also managed to raise an impressive chunk of capital as it seeks to use its specifically targeted CAAR T cell products to transform the lives of patients with autoimmune diseases. In 2019, the company raised $50 million in series B funding to increase the company’s total funding to $88 million. This has been used to develop CAAR-T therapies.
Founded in 2017 and based in Philadelphia, US, the biotechnology company uses a selective B cell Ablation (CABA™) platform, in combination with Cabaletta’s proprietary technology, to selectively bind and eliminate only specific autoantibody-producing B cells while sparing normal antibody-producing B cells, which are essential for human health. The CEO Gwendolyn Binder aims to deliver deep and durable - and potentially curative - gene responses. Recent clinical trials showed no dose-limiting toxicities or clinically relevant adverse events with patients receiving 100 million DSG3-CAART cells, a five-fold higher dose than the initial cohort.
Orgenesis is another US company, with a POCare Platform built on three components to accelerate the development and production of advanced medicines, unlocking the potential of cell and gene therapy for all.
Founded in 2008 and based in Maryland, Orgenesis Inc overcomes conventional processing challenges by enabling sterile, scalable onsite processing for a pipeline of advanced therapies.
In 2019, over 1,000 cell and gene therapy-related clinical trials took place. While many of these trails were successful, most biotechnology companies lack the power and expertise to scale the therapies’ development and manufacturing. This makes progress slow and costly. Orgenesis works with these companies to overcome existing challenges of CGT production with process development, manufacturing systems, logistics and regulatory expertise. The biotech also conduct collaborative research, clinical trials, process development and commercial production with growing networks of hospitals and healthcare providers.
Vered Caplan, the CEO, says the company is committed to bringing advanced therapies to market effectively. Orgenesis recently announced a new collaboration with biotech firm Savicell Diagnostics to help accelerate the development and manufacturing of its cell and gene therapy platform. The agreement allows Orgenesis to use Savicell’s immunometabolism platform to help advance its own platforms on several fronts. This has motivated rapid growth, with 2Q revenue rising from $1.75 million to $10.5 million in 12 months.
Unlike the four other companies mentioned, Deep Genomics is using artificial intelligence (AI) to build new life-saving genetic therapies and treatments. Founded in 2014 and based in Toronto, Canada, Deep Genomics aims to change the future of biology with biologically accurate AI technology.
Finding therapies requires mining RNA biology data, but the vast nature and complexity of the data make standard approaches overwhelming, costly and slow. Deep Genomics places the RNA biology data into the AI workbench which untangles this` complex data, identifies the novel targets and evaluates thousands of possibilities to identify the best therapeutic candidates. The AI system saves valuable time and money, in turn, making treatment more accessible to a wider audience.
Brendan Frey, the CEO, seeks to program therapies for any gene and genetic condition dramatically improving people’s lives with every advancement. The therapeutics company recently announced the closing of a $180 million series C financing round further validating the significant advances in the AI discovery platform and the growth of the proprietary preclinical pipeline.
The global cell and gene therapy manufacturing services market is expected to grow by 12.4 per cent in the next five years, reaching a market value of $13.8 billion by 2026. With the markets continued growth and development the gene and cell therapy industry, this is an exciting sector to be a part of. It’s a privilege to serve the market with my contacts and recruitment services.
If you’d like to learn more about these companies or the talent market with the biotechnology space, please don’t hesitate to contact me. My email is Spencer.Knight@lifesci-cm.com.
Small molecules are now able to target previously ‘undruggable’ conditions, such as cancers and neurological disorders, I discuss how we got here and what it means for the market. Click to read more.
In part two of this discussion, Dennis and I discuss the key barriers for entry for companies expanding into new territories and how to overcome these, whether you're a start-up or established company entering a new market. Click to listen.
In this final part, Dennis and I discuss new technologies to watch in the liquid biopsy market, as well as insights into innovation from behind the scenes at Predicine. Click to listen now.
Predicine With a background in research, pharma and commercial, Dennis gives insight into the true value of liquid biopsy. Click to listen.