CPHI Online Newsletter: Issue 42

The?CPHI Online?Weekly Newsletter brings you the latest bite size?#pharma?news straight to your LinkedIn inbox.

Want to keep your finger on the pulse?

? Look no further ??subscribe here.

So? What are we looking at this week?

It's the peak of summer - or if you're based in Western Europe, we might be asking ourselves - what summer? Today I think may have been the first day of blue skies in almost six weeks. Regardless the cogs are whirring and it's been a very busy week in the pharma world.

At CPHI, our editor Lucy sits down with Sheila Ann Mikhail, the co-founder and CEO of AskBio to talk about all things gene therapy, it's cross over into AI and how to be an informed innovator when picking your manufacturing partner.

But firstly - to break it down and set some context - what is gene therapy? As Pfizer defines, gene therapy is a new generation of medicine?where a functioning gene is delivered to a targeted tissue in the body to produce a missing or nonfunctioning protein. By using genes as medicine, the underlying cause of a disease can be targeted at the cellular level, potentially with just one treatment. Pfizer has also identified gene therapies as a key pillar of their Rare Disease strategy.

"I want to really outline the state of the gene therapy research industry that is at a real inflection point now, in particular how to make these therapies more affordable and accessible to larger populations." - Sheila Anna Mikhail

Gene therapies already have their uses throughout pharma but to advance these forward it's crucial to work out how they can be used to treat pathway diseases. Bringing gene therapy modalities to patients successfully remains a challenge.

A 2022 McKinsey article How AI can accelerate R&D for cell and gene therapies brings the topic of AI into the picture - "while AI applications are taking off in the wider biopharmaceutical R&D context, companies are only starting to explore how to apply their potential to Gene Therapies, or CGT (Cell and Gene Therapies).

It continues "There is significant untapped opportunity in the industry to scale AI within the CGT value chain. Biotechnology companies enabled by machine learning (ML) that focus on novel modalities are still rare." (https://www.mckinsey.com/industries/life-sciences/our-insights/how-ai-can-accelerate-r-and-d-for-cell-and-gene-therapies)

Applying AI to R&D novel therapeutic modalities brings to light three main concerns:

1. Limited experimental data avaliable and expensive data generation
2. Functional complexity
3. Separation between wet-lab and in silico research (in silico requiring teams to work alongside each other in "pods" as opposed to together

Back to CPHI, and Lucy continues the article exploring Sheila's mindset on the supply chain and accelerated innovation:

"Standardisation across the industry is key to process acceleration and that's something we all need to work toward. You want to develop a process that can be done once and then retrofitted to fit elsewhere." - Sheila Anna Mikhail

On comments around supply chain and CDMOs Sheila maintains "If you don’t control the supply chain carefully, you get a lot of contamination in your products – which then impacts the quality of the final product. CDMOs are still incredibly useful, but you have to be a knowledgeable buyer of CDMO services and an invested consumer."

And where better to listen to Sheila than in person at CPHI.

Come and experience the biggest pharmaceutical event in Europe - and be amongst those spearheading the way forward in the future of pharma.

?? ?? ???Aaaaaand that's a wrap! We'd love to stay and chat but we know you have work to do and so do we - find our?latest articles here?and stay tuned as usual for more.