Bringing you insight from our in-house sector experts, the ramarketing review delivers vital industry knowledge straight to your inbox! Each month we feature a different ramarketing expert to deliver headline highlights.
Joao joined ramarketing as Bio Sector Lead and has over 15 years of experience in the biomedical sector in the US, UK, and EU.
Joao is a former medical doctor and a Harvard-trained scientist, with a Ph.D. in Molecular Medicine and Oncology. Having received prizes for his academic work and achievements from prestigious biomedical organizations he has a vast knowledge of the pharma and biotech space. As Bio Sector Lead, Joao contributes actively to the successful delivery of projects through technical know-how, content review, messaging insights, and market knowledge to ensure our work is high quality, industry-specific and world-class.
The gene therapy field has faced a few safety, clinical and regulatory setbacks over the past couple of years, and has seen the FDA approving only four gene therapies in the five years prior to 2022. However, with dozens of small biotechs jumping into the field over the last decade, we are starting to see more and more approvals, including at the back end of last year, Bluebird’s Skysona and Zynteglo, CSL’s Hemgenix, and the first allogeneic T cell therapy product, developed by Atara. In my view, 2022 marked a significant reignition in the field of advanced therapies.
Looking ahead, close to twenty cell and gene therapies are set for a regulatory decision this year, and at least five gene therapies for rare diseases are expected to be approved in the US. This includes the first CRISPR gene editing medicine from Vertex/CRISPR Therapeutics, with its submission to the FDA imminent and approval expected later this year. A potential cure for the blood disorders sickle cell anaemia and beta-thalassemia, it has a convoluted name, exagamglogene autotemcel, and it will be the first gene editing therapy based on the Nobel Prize-winning CRISPR technology.
Behind Vertex/CRISPR assets, the C>/RNA therapy pipeline boasts just under 4000 assets in development, and the close to double-digit pipeline growth really demonstrates that this is a space set for many breakthroughs to come, particularly in the dominating fields of oncology and rare diseases.
The prolonged downturn and economic headwinds faced by biotechs in the last 18-24 months must certainly be testing their resilience. The public markets are likely to reopen next year, or at best, later this year, and access to private capital remains a challenge, with investors continuing to be selective about their capital allocations. Global cell and gene therapy investments fell quite a bit from the c.$20 billion per year of the golden 2020-21 years to c.$13 billion last year, in line with pre-pandemic levels. This means that many C> biotechs, both small and large alike, have had to downsize their workforce and rethink their pipeline prioritization strategies. These include, for instance, BioMarin and Editas Medicine, with the latter cutting back on several clinical-stage therapies. This may mean that ultra-rare diseases, which typically represent smaller commercial opportunities, may end up on the back burner.
To make matters worse, the collapse of Silicon Valley Bank (SVB) means that for many early-stage private biotechnology companies, access to funding to sustain critical drug development operations became even scarcer. The full extent of the implications of the collapse is yet to be known, but the reality is that about 50% of US biotech companies banked with SVB will now have to turn to alternative, perhaps somewhat less biotech-friendly, lenders.
In the coming months we are certainly going to see restructuring, consolidation, M&A, and collaborations that benefit from the scale and efficiencies large pharma offers, and unfortunately some failures as well.
How the British arm of the Silicon Valley Bank saved the British biotech sector from collapse, but the remaining fallout could have lasting effects on the sector in years to come.
Tech start-ups are looking to understand the damages done by the collapse of SVB, and how it can affect them and others looking to break the market in the near future.
With the drug development timeline being a complicated process, what can CDMOs do to help smaller biotechs meet the timeline goals to allow them to achieve success, in the ever-changing bio and pharma space?
What is on the horizon for the Pharma and Biotech space? How do the latest market trends affect your business going into Q2 of 2023?
Stay tuned for more ramarketing review installments – who will next month’s guest expert be? You’ll have to be subscribed to our mailing list to find out…
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