Big Challenges Hit Cell and Gene Therapy Leaders as Industry Shifts Focus

Solving the Commercial Puzzle to Succeed in Cell and Gene Therapies

This Year's Challenges to CGT

This year witnessed two leading examples of the challenges faced by the cell and gene therapy space, named Bluebird Bio and Sarepta Therapeutics. The two leaders, despite having products on the market and a high value of over $10 billion, turned out to be a living nightmare in recent months. Sarepta’s market dropped instantly to around $1 billion, while Bluebird was taken over for $50 million by two equity firms. Sarepta’s market and bluebird products are running unfavorably. Bluebird’s new owners, consisting vast Carlyle Group, run the operations.

These two companies' fate spotlights the ongoing hurdles faced in the CGT area. At the Bio Convention in Boston this summer, at the Biospace moderated panel, the experts discussed what needs to happen to initiate a new class of medicines progressively for a longer period. The discussion further explored the epicenter of manufacturing and its regulatory factors that might cut the prices and extend access, and the positive impact of tariffs. While the field advances at the forefront, everyone’s attention is on the companies that introduce new therapies to the needy patients.

On the other hand, the next-generation technologies are immensely entering the manufacturing side of the CGT environment in terms of production and its regulatory aspects. Foster and other penalists at Bio agreed to this point, and the global industry is now realizing and recognizing the importance of this area of the equation.

Changes in Manufacturing and Regulation

In July, the UK’s medicines and healthcare products regulatory agency (MHRA) introduced a new regulatory framework for manufacturing. The guideline series published during the launch of the framework, the regulator initiated a discussion on how to execute new modular manufacturing and point-of-care legislation. This received approval in January, along with the commercialization authority for the application of good manufacturing practices in incorporated facilities and designation.

The distributed manufacturing is an advantage to veins to shorten to vein time to receive patients, affecting illnesses that can be treated fast without freezing cells for shipping.

Statements of Experts

CEO of Oribiotech, Jason Foster, said, “I think the purchase of Bluebird by Carlyle is exciting. The commercial success is still questionable, so the interesting part of this case study would be whether we can select the failed product or company or set of products as something that can be commercially acceptable with the next generation of technologies?”