Cell and gene therapy exits from Sanofi and AvroBio

Sanofi will focus its cell therapy strategy on allogeneic products, terminating its partnership with Sangamo. Meanwhile, disappointing trial results have driven AvroBio to abandon its lead gene therapy candidate for Fabry disease.

The cell and gene therapy space continues to evolve but two announcements demonstrate the vicissitudes that linger over this burgeoning sector.

Firstly, French Pharma giant Sanofi has ended a long-term hemoglobinopathies partnership with genomics firm Sangamo Therapeutics.

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“In its notice to Sangamo, Sanofi indicated that its termination relates to Sanofi’s change in strategic direction to focus on allogeneic universal genomic medicine approaches rather than autologous personalized cell therapies,” Sangamo wrote in an SEC filing dated December 30 2021.

Sanofi entered the allogeneic, or off-the-shelf, cell therapy space in November 2020 through the €308 million ($350 million) acquisition of Kiadis. “We believe the Kiadis ‘off the shelf’ K-NK cell technology platform will have broad application against liquid and solid tumors, and create synergies with Sanofi’s emerging immuno-oncology pipeline,” the firm said at time.

Meanwhile, it has been scaling down other advanced therapy programs, dropping a Friedreich’s ataxia gene therapy candidate collaboration with Voyager Therapeutics in 2019, and selling back the rights to a potential gene therapy for GUCY2D-associated Leber Congenital Amaurosis to Atsena Therapeutics.

Sanofi’s involvement with Sangamo dates back to 2018 when it acquired Bioverativ for $11.6 billion. Bioverativ, a hemophilia spin-out from Biogen, teamed with Sangamo in 2014 to develop autologous cell therapies for beta thalassemia and sickle cell disease.

The agreement also saw Sangamo grant Bioverativ/Sanofi an exclusive, royalty-bearing license to use its tech, including its zinc finger protein platform, for researching, developing, manufacturing and commercializing licensed. Sangamo received $20 million up front with potential milestone payments of up to $276.3 million. However, just $13.5 million of this has been achieved to date.

“Following the Termination Date, Sangamo will not be entitled to receive any further milestone payments or royalties from Sanofi,” Samgamo wrote. “As of the Termination Date, Sanofi will have no further obligations to develop or to fund the development of any Collaboration Research Programs under the Collaboration Agreement.”

AvroBio’s disappointing results

In other news, Massachusetts-based firm AvroBio announced on January 4 it is deprioritizing its Fabry disease program – its lead gene therapy candidate – following disappointing trial results.

“Previously reported data from 13 patients treated across our three clinical-stage programs have shown durable engraftment out 9 to 54 months,” said CEO Geoff MacKay. “It is the new data from the five most recently dosed Phase II FAB-GT patients that are discordant with these other data and show variable engraftment.”

He added: “In addition, the last 12 months have presented multiple challenging market and regulatory dynamics for our Fabry disease program, which would now be exacerbated by a meaningfully extended development timeline.”

The candidate AVR-RD-01 has been removed from AvroBio’s pipeline.