The clinical development of next-gen Cell and Gene Therapies (CGTs) shows substantial potential in the healthcare space. We summarize the recent rapid advances and their impact.
Cell therapies have evolved with multi-generation CAR-Ts, primarily targeting hematological malignancies and rare indications. The focus is now shifting towards off-the-shelf therapies and more innovative technologies such as NK Cells, iPSCs, Bi-specific DART, Bi-specific γδ T-cell engager and others.
In gene therapy, the delivery modality space has been dominated by Adeno-associated virus vectors. Lentivirus vectors are now stepping into the picture. The trend with gene therapies is towards customizable gene editing with tools and techniques such as CRISPR, TALEN and ZFNs.
While CGTs are at the forefront of healthcare innovation and among the fastest growing therapeutic modalities, the area warrants more scientific and clinical research to answer concerns on long-term efficacy, safety, immunogenicity, complex manufacturing and premium pricing, to name a few.
Furthermore, regulatory bodies in the US and EU have issued several guidelines for preclinical testing, product development and clinical trial design. Developers are engaging with regulators more frequently from an early stage of development to minimize regulatory setbacks.
It is imperative for pharma companies to collaborate with agile and intelligent knowledge providers and consulting partners to power their go-to-market strategy.