CSL dedicated to Hemgenix as it turns back on lenti-based gene therapies

The research and development (R&D) facility in Pasadena, California will close its doors by the end of January 2025 as part of an advanced-therapy pipeline restructure that will deprioritize ex vivo lentiviral-based gene therapies. The facility was added to CSL’s network through its $91 million acquisition of Calimmune in 2017, which also added the abandoned program CAL-H, an ex vivo hematopoietic stem cell (HSC) gene therapy.

“We have conducted a strategic evaluation of our R&D footprint to support this change and have identified opportunities to capitalize on a strategic and technical convergence with our sa-mRNA [self-amplifying messenger RNA] capabilities and leverage synergies across the technologies/platforms,” the firm confirmed to BioProcess Insider.

“As such, CSL will move our cell and gene therapy (CGT) research and development activities to Waltham, Massachusetts and close our Pasadena-based operations.”

Globally, CSL employs more than 2,000 R&D employees. The Pasadena site hired more than 50 employees following an expansion in 2020, and while some positions will relocate to Waltham, about 30 people will be made redundant, the spokesperson said.

CSL Behring entered the sa-mRNA space through a $200 million collaboration and license agreement with Arcturus Therapeutics in 2022. The company gained access to Arcturus’s sa-mRNA vaccine and lipid nanoparticle (LNP) platform technologies, that, according to the firm, tackle delivery problems typically associated with viral vectors and existing lipid vector technology by capturing therapeutic nucleic acids and safely moving them to target cells using an endocytosis process.

Following Calimmune, CSL grew its presence in CGTs through a $450 million upfront collaboration in 2020 with uniQure, when it attained global rights to a hemophilia B gene therapy that 18 months later won US approval under the brand Hemgenix (etranacogene dezaparvovec).

Importantly, Hemgenix is an in vivo adeno-associated viral-based (AAV-based) gene therapy, rather than ex vivo lentiviral-based. Thus, CSL will still focus on gene therapies as part of its wider strategy.

“We are excited about Hemgenix as an important treatment option for appropriate patients living with hemophilia B and are proud to have delivered, with our partner uniQure, the first gene therapy for patients living with this serious condition,” the spokesperson told us.

“Our dedication and focus on Hemgenix is steadfast, and our decision to move our genetic medicines capabilities to Waltham has no impact on Hemgenix.”

Hemgenix is manufactured at a Lexington, Massachusetts site formally owned by uniQure. Contract development and manufacturing organization (CDMO) Genezen acquired the site earlier this year for $25 million.

Across the board, the gene-therapy sector has suffered from slow rollouts and payer problems. While CSL does not publicize individual product sales, the firm said in its first fiscal year post-launch, Hemgenix – which has a list price of $3.5 million – contributed to a 10% revenue increase within a hemophilia portfolio that totaled $1.3 billion. The firm also noted some success in inking reimbursement approvals in the US, as well as in the EU, UK, Switzerland, and Canada.