Internal innovation engines established by top pharma
Expertise and innovation in emerging fields of science such as cell and gene therapies had been predominantly associated with biotech, however, more and more of top pharma are investing in developing their internal capabilities either organically or inorganically. Novartis Gene Therapies focuses on AAV (adeno-associated virus)-based therapies that deliver functional genes into human cells for neurology and ophthalmology and CRISPR-based technologies that manipulate DNA for haematology and ophthalmology. Set up in 2013 and bolstered by the $8.7bn acquisition of AveXis and spinal muscular neuron gene therapy, Zolgensma for spinal muscular atrophy in 2018, further significant investments were made including a $3bn license for Voyager Therapeutic’s AAV capsids. Roche has its Pharma Technical Cell & Gene Therapy unit and has made large investments in the space, including a $4.8bn acquisition of Spark Therapeutics in 2019 with Luxturna, a one-time product, in approved for retinal dystrophy, a rare form of blindness, being a key part of the deal. Roche has also utilised partnerships to bulk up its capabilities in neoantigen-targeting T-cells for oncology (Adaptive Biotechnologies), engineered T-cells for solid tumours (Arsenal Biosciences), CAR-T therapy for haematological cancer (Poseida Therapeutics), allogeneic approaches start with induced pluripotent stem cells (Adaptimmune), and Regenerative cell therapies for eye diseases (Lineage Cell Therapeutics).
