Sanofi says it is looking to resolve the “patchy” accomplishments in the gene therapy space through a new specialized unit that will leverage technologies from its vaccine division.
Sanofi has dabbled in the cell and gene therapy over the past decade but is yet to make its mark. Speaking at the JP Morgan Healthcare Conference this week, CEO Paul Hudson highlighted the importance of the sector going forward but admitted industry’s success in the sector has “been patchy.”
He told delegates that “even when there have been effectively miracles they have not always been readily adopted.” The handful of approved therapies have often suffered from high costs of goods (and high prices), patient access and manufacturing problems.
Sanofi spoke at the 39th Annual JP Morgan Healthcare Conference. Image: iStock/danielvfung
“We understand as a team what it’s going to take to address cost of goods and allow for pricing to drive a broader access for patients that would ordinarily be excluded and we don’t want that.”
Big Pharma and regenerative med
Regenerative medicine represents one of the most exciting developments in the healthcare space. As more and more cell and gene therapies reach commercialization, Big Pharma companies are increasingly adding candidates to their pipelines through M&A activity and investing in next-generation technologies.
Gilead acquired Kite Pharma in 2017 adding CAR-T therapy Yescarta (axicabtagene ciloleucel), for example, while Roche bought Spark Therapeutics for $4.3 billion in 2019, adding Luxturna (voretigene neparvovec), a one-time gene therapy for an inherited retinal disease approved by the US Food and Drug Administration (FDA) in December 2017. And just last month, Eli Lilly kick started its cell and gene therapy strategy through the $880 million acquisition of Prevail Therapeutics.
Meanwhile, fellow big pharma firms Novartis and Pfizer have both invested hundreds of millions of dollars in their cell and gene therapy networks over the past couple of years.
But currently Sanofi’s pipeline only includes two ex vivo ZFN gene-edited cell therapies being investigated in rare blood disorders. And over the past year the firm has dropped several gene therapy programs with partner Voyager Therapeutics, and sold back the rights to a potential gene therapy for GUCY2D-associated Leber Congenital Amaurosis to Atsena Therapeutics.
Leveraging Sanofi Pasteur
Despite this, global head of R&D John Reed said he is positive Sanofi will be a major player in the next-generation of cell and gene therapies, playing off its early attempts to play in the space while utilizing the technologies developed within its vaccine division.
“The company through its Genzyme heritage had worked in gene therapy well over a decade ago and put a couple of AAV [adeno-associated virus] vectors into the clinic for ophthalmology. But it was never really embraced and supported in the way that it needed to,” he told JP Morgan delegates.
“The manufacturing process for making viral-based gene therapy is very similar to some of the technologies that are used in our vaccines group at Sanofi Pasteur […] They use very, very similar technologies to produce viral and very effective vaccines at scale, and so, leveraging those as internal competencies, we now have organized our gene therapy into an end-to-end unit, we call the Genomic Medicines Unit.”
And within the past couple of months, Sanofi has begun construction of a €15 million ($18 million) viral vector facility in Lyon, France to support its gene therapy strategy. And in November, the firm made a major leap into the cell therapy space through the $358 million acquisition of Kiadis.