24/7 BIOPHARMA - issue 1 / October 2024

RECIBIOPHARM

Advanced therapy medicinal products (ATMPs) have taken centre stage in the biopharma industry over the last decade, offering groundbreaking treatments for rare and difficult-to-treat diseases. With over 2,000 products currently in development, the gene therapy segment, in particular, is experiencing substantial growth [1] . Gene therapies offer potentially life changing treatments for difficult-to-treat diseases but are also consistently ranked among the most expensive therapies in the world. At $4.25 million for a one-time treatment, the most expensive drug on the market is Lenmeldy (Orchard Therapeutics), a gene therapy for metachromatic leukodystrophy (MLD) [2] . More gene therapies are emerging that show promising and repeatable efficacy for a range of diseases. However, their high price point can create a significant cost barrier. To address this challenge, adeno-associated virus (AAV) therapy developers are increasingly seeking new and flexible solutions for gene therapy manufacturing while maintaining product safety and efficacy. By developing the next generation of AAV manufacturing platforms, developers and manufacturers are helping to lower AAV product costs and deliver more affordable gene therapies.

However, the AAV production process is complex, with multiple intricate steps from plasmid production to fill and finish, and there is no standardised method or platform for the development and manufacturing of these therapies. This means flexibility is key in AAV manufacturing. By focusing on flexibility when developing an AAV manufacturing platform, gene therapy manufacturers can overcome production challenges to meet industry needs and improve the affordability of these innovative medicines. Building flexibility into an AAV manufacturing platform can accommodate the unique characteristics of different AAV products and therapeutic targets and helps with optimising product yield, quality, safety and efficacy. Considerations for building flexibility into manufacturing processes include: - GMP plasmid access Plasmids are essential in gene therapy development and manufacturing, impacting product quality, yield and project timelines. Access to GMP-grade plasmids is critical and is a well-recognised bottleneck in AAV production. Essential elements of flexible AAV manufacturing platforms

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TWENTYFOURSEVENBIOPHARMA Issue 1 / October 2024