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The Winter 2022/2023 issue of the Drug Discovery World Magazine is accompanied by an exclusive guide on cell and gene therapy (CGT). DDW Multimedia Editor Megan Thomas comments. 

Putting this guide on cell and gene therapy (CGT) together has not only been an exercise in realising the seemingly boundless opportunity within the sector, where words like ‘cure’ and ‘cancer’ can be used optimistically in the same sentence, but it has also shone a light on just how many people – scientists, researchers, business leaders, lab technicians, managers – play a role in its development. This is a community working towards a common goal.

Collaboration can come in many shapes and sizes. This can come in the form of events and forums such as the ISCT Commercialization Signature Series. ISCT also offers a joint biomanufacturing training programme through a partnership with Engineering Research Center for Cell Manufacturing Technologies (CMaT), which is funded by the US National Science Foundation. Later in this guide, George White shares insight into Cytiva’s industry courses and we hear from Phacilitate’s Ryan Leahy ahead of Advanced Therapies Week 2023.

This is not an industry that can evolve in isolation. Thankfully, this is well-known, evidenced through the collaborations taking place across the sector. GenScript and Avectas, for instance, have joined biotech forces for a research project to streamline manufacturing of non-viral CRISPR gene editing components, with the goal of improving the editing efficiency and cell viability of non-viral based cell therapies. Meanwhile, CGT Catapult partnered with the UK Dementia Research Institute (UK DRI) to help advance the development of adeno-associated virus (AAV) based gene therapies for dementia. This only just scratches the surface.

Despite how innovative this work can feel, the reality is that the first therapy was only approved in 2017 and the number of approved treatments remains small. Of the 50-plus drugs approved by the FDA in 2021, only two were cell or gene therapies1. That said, there are plenty of developments which encourage optimism. With ‘why’ and hopefully ‘how’ covered, the next questions are: What is underway? What does the future hold? Where are the developments? When will we see deliverables? These are the sorts of questions the experts across this guide try to answer.

To name a few, Emulate has launched new AAV transduction application for the liver-chip that enables gene therapy researchers to test the delivery efficiency and safety of AAV vectors in a validated, human-relevant model of the liver and get results in weeks, not months, as with animal models. SiSaf, an RNA delivery and therapeutics company, is initiating the US FDA Regulatory process to obtain an Orphan Drug Designation for a therapeutic for patients with a rare genetic skeletal disorder. In late November, uniQure received FDA approval from for HEMGENIX (etranacogene dezaparvovec-drlb), a one-time gene therapy for the treatment of adults 18 years of age and older living with haemophilia B – the first of its kind. At Lonza, two CGTs have reached commercial approval: ZYNTEGLO, for the treatment of transfusion-dependent beta-thalassemia; and SKYSONA, for the treatment of early, active cerebral adrenoleukodystrophy.

According to McKinsey, in 2024 alone, up to 21 cell therapy launches and as many as 31 gene therapy launches—including more than 29 AAV therapies—are expected2. Currently, there are more than 1,000 ongoing clinical trials for CGTs (registered with ClinicalTrials.gov); the majority of them remain in Phase I and II and are thus not within immediate reach of commercialisation1. Regardless, the proportion of early-stage CGT trials still represents 10% of all clinical activity, suggesting the only way is up.

References

  1. https://www.cellandgene.com/

  2. https://www.mckinsey.com/industries/life-sciences/our-insights/eight-imperatives-for-launching-cell-and-gene-therapies