Advanced Therapies 2024

06 Jun

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The Advanced Therapies Congress brings together speakers from across the entire value chain of cell and gene therapy development; forward-thinking pharma, biotech and startup companies, researchers, clinicians, academics, HTAs, payers, regulators.

At my recent panel passion in BISNOW (7th March 2024) Life Sciences and Innovation Real Estate Annual Conference at 1 Triton Square in London, I suggested that cell & gene therapy is dominating the life sciences research in 2024 and beyond. I also mentioned that if the last century was all about chemistry, this century is all about biology.

Coincidently, within two weeks (19-20 March 2024), I had an opportunity to attend the Advanced Therapies conference in ExCeL London which was all about cell & gene therapies & ATMPs (Advanced Therapy Medicinal Products). The packed two day event was boasted by global experts in the field delivering keynote sessions, 100+ exhibitors, multiple parallel tracks on key topics (cell therapy, gene therapy, immunotherapy, innovation, clinical development, vector manufacturing, regulation, clinical development & more), poster presentation, networking and round table conversations.

It was fascinating!

I have worked in small molecule drug discovery & development for several years. The world of large molecules unfolding and evolving was indeed fascinating.

I met may knowledgeable and passionate delegates, witnessed latest instrument innovations, listened to real issues and learnt a lot.

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The key takeaways are as follows:

  • Advanced therapy field, ATMP (Advanced Therapy Medicinal Products) is advancing very fast. All the stakeholders are trying to keep up-to-date with the advances.
  • Advanced therapies started with rare diseases and on small patient population, the challenge is to bring them to other disease areas, cost effectively.
  • Often the therapy is single dose treatment and available in developed countries. Another challenge is to bring treatments to low-medium income countries with limited resources but more patient population.
  • A big gap between academic research and industrial commercialisation exists due to costs of GMP manufacturing. A bottleneck due to costs of setting up, operating and maintaining a GMP facility. A CDMO (Contract development and manufacturing organization) which provide outsourcing options to biotech and pharma companies claimed that they fill this gap by providing GMP services.
  • Effective technology transfer from basic research to CDMOs could take time and vary from technology to technology.
  • Advice from one of the experts - As the processes are still developing, chasing a perfect process may not be a good idea but a continuous process improvement approach might work better. Understanding the product could help get the right process was another speaker’s advice.
  • Move from open processes (on bench) which might need grade B clean room facility to closed system (bioreactors) could help. The closed system will downgrading the GMP facility need from B to C hence reducing costs.
  • Cost is a tight rope. A lot of discussion around access, re-imbursement with a general agreement that all patients deserve a good treatment.
  • Raw material availability, quality and supply logistics consideration very important.
  • Quality control theme came up with a few times and a lack of resources (personnel) mentioned.

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  • Regulatory bodies, their involvement, education and the role of KOL (Key Opinion Leaders) was mention with importance of education.
  • In clinical development theme, the Great Ormond Street Hospital medical practitioner highlighted issues in patient recruitment for clinical trials, potential issues, mitigation measures and role of social media in patient recruitment was interesting (spreading of misinformation as well as good publicity).
  • I attended a round table discussion about ‘ 3D cultures to Cell Therapies’ with an interesting mix of start-up founders, academia and industry professionals. Hydrogel technology to encapsulate single cells and issues encountered were discussed and whether other biomaterials that can be used as alternatives. In this session, gap between academic and industrial research (due to basic, exploratory research to commercial product) was discussed. I suggested if academic labs should have more GMP labs (expensive but government funding could benefit).
  • It was also recognised that the advanced therapies research occurs in specialised centres by trained staff and continuous training is essential.
  • In Advanced Therapies, Science and Business go hand-in-hand was mentioned by a speaker which I agree. New therapies are great but commercial viability could be an issue and a great challenge to solve for the scientific community. A healthy discussion about access even if not commercially viable by cross-border treatment by bringing patient to product followed. But the difficulty in bringing large patient population from low-medium countries to developed countries was raised.
  • In Gene editing track, what is Cas9 and what does it do was discussed? An enzyme called Cas9 acts as a pair of 'molecular scissors' that can cut the two strands of DNA at a specific location in the genome so that bits of DNA can then be added or removed.

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One of the other highlight was meeting our client, Refeyn as one of the exhibitors at the conference. They reported a huge interest in their technology by the conference delegates. Refeyn's mass photometry technology can rapidly quantify the mass of single molecules in solution, without labels.

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It was also great to catch-up with one of our key science developer contacts and a cleanroom supplier.

I learnt new terminologies – Mechanobiology (where physical stress is applied to cells to assess their response or state. To make me understand the technology, the engineer explained as if while buying an avocado how one would press it to check if ripe; LNP, Lipid Nano Particles, ISPC – Induced Pyropluoric Stem Cells to name a few.

Overall, it was a great learning experience and I look forward to attending the next year’s congress. At Bulb, we pride ourselves on focussing on clients’ Science First and one of the keynote speakers summed it saying SCIENCE WILL ALWAYS PREVAIL.