CSL Behring LLC

CSL Behring is closing a Pasadena, CA-based R&D site and moving the operations to Massachusetts as part of a larger repositioning of the company’s gene therapy research. A spokesperson for the company said Monday that it will deprioritize ex vivo lentiviral-based gene therapy work after finding overlap with existing sa-mRNA capabilities. The news was first reported by Fierce Biotech on Monday. The Pasadena facility, which CSL Behring gained from its 2017 acquisition of Calimmune, is set to close by the end of January 2025. The spokesperson said that some employees will make the cross-country move to Waltham, MA, but that 30 won’t be kept on. The Pasadena space was the company’s first in-house cell manufacturing outpost. Investments had been pouring into the site through 2021, with the most recent expansion adding nearly 3,100 square feet and additional office and lab facilities. Previously, from 2018 to 2019, CSL Behring added a Good Manufacturing Practice facility plus two labs. Reconsidering some of its gene therapy research comes as the company is looking to build demand for its hemophilia B treatment, Hemgenix. CEO Paul McKenzie said in August that 12 patients were treated in the 2024 fiscal year, which ended in June, with a “handful or so” treated in July. The one-time therapy’s late 2022 entrance onto the market was polarizing, thanks to its $3.5 million list price in the US. In June 2023, BioMarin snagged FDA approval for its one-time hemophilia A treatment, Roctavian. That $2.9 million treatment has had similarly slow uptake, contributing to cost cuts and layoffs.

CSL Behring is deprioritising its ex vivo lentiviral-based gene therapy platform and closing its hub for cell and gene therapy R&D in Pasadena, California as it explores other gene therapy approaches, a company spokesperson confirmed to FirstWord on Monday.The closure, set for the end of January 2025, comes as CSL's sole marketed gene therapy Hemgenix (etranacogene dezaparvovec-drlb) continues to face commercial hurdles.The spokesperson said CSL will consolidate its cell and gene therapy R&D operations in Waltham, Massachusetts, as part of a broader strategic evaluation of its R&D footprint. They didn't specify how many jobs would be affected, but said approximately 30 employees, primarily in Pasadena, will be leaving the company, though some staff have accepted relocation offers to the Waltham facility.CSL acquired the Pasadena site through its purchase of Calimmune in 2017 for an upfront payment of $91 million.The pivot comes amid industry scepticism about the commercial viability of certain gene therapy approaches. Recent expert feedback compiled by FirstWord indicates that while Hemgenix, which uses an in vivo method of gene transfer, has demonstrated clinical efficacy in haemophilia B, the therapy faces substantial financial and logistical barriers to widespread adoption, with physicians awaiting longer-term durability and safety data. See – Spotlight On: KOLs find a new balance in haemophilia B."This strategic shift will enable us to focus our resources on areas showing the most promise while enabling our teams working on the leading edge of genetic medicines — such as mRNA and gene therapy — to work closely together," the spokesperson said, adding that the company sees opportunities to capitalise on "strategic and technical convergence" with its self-amplifying mRNA capabilities.The closure of the Pasadena site, which CSL acquired along with Calimmune's Select+ and Cytegrity platforms for the development of ex vivo haematopoietic stem cell gene therapy, marks a departure from the company's initial gene therapy strategy. When CSL purchased Calimmune, then-CEO Paul Perreault had highlighted the acquisition as an "important step" in executing the company's strategy for sustainable growth, particularly in addressing commercialisation challenges in stem cell therapy.

MELBOURNE – 24 October 2024 – Two Australian scientists have each been awarded CSL Centenary Fellowships, valued at $1.25 million over five years. The Fellowships were presented at the Australian Academy of Health and Medical Sciences Annual Meeting on Thursday 24 October 2024 in Adelaide. Lung cancer is now our deadliest cancer, despite the reduction of smoking in recent decades. Twenty-five per cent of people with lung cancer have never smoked. Over the past 12 years, Dr Clare Weeden has investigated why lung cancer is on the rise in cities around the world. She has shown that we all have potentially cancerous cells in our lungs which can be activated by repeated exposure to cigarette smoke or urban pollution. The $1.25 million CSL Centenary Fellowship has enabled Dr Weeden to return from the Crick Institute in London to establish her own research laboratory at WEHI in Melbourne. She plans to identify how the chromatin that packages up our DNA is changed by inflammation in lung cells. Then she will investigate how these cellular changes initiate cancers and how cells then become resistant to targeted therapies. Dr Weeden’s ultimate career aim is to determine if abnormal lung cell states are reversible. Dr Qi Zhang is investigating the fundamental processes by which our cells turn genes on and off as they change identities, for example as stem cells develop into mature cell types. She hopes to learn how these processes can break down and lead to cancer and other diseases. Dr Zhang is a team leader at the South Australian immunoGENomics Cancer Institute (SAiGENCI), University of Adelaide. “We want to know what’s happening with the packaging of our DNA in a healthy cell,” she says. “Then we want to know what is going wrong in a cancer cell – when it loses its identity.” Using the CSL Centenary Fellowship, Dr Zhang hopes to generate fundamental knowledge that researchers around the world can use to develop new drugs to tackle epigenetic misregulation in cancers. CSL Head of Research and Chief Scientific Officer Dr Andrew Nash said, “Dr Zhang and Dr Weeden are both making fundamental discoveries about how normal cells develop and how that development can go wrong leading to cancer and other diseases.” “With the support of their CSL Centenary Fellowships, their research will open up paths to new kinds of treatment for cancer and developmental diseases,” he said. “The CSL Centenary Fellowships aim to support leading mid-career Australian researchers like Qi and Clare by providing funding stability to enable the delivery of innovations that could transform medicine for patients living with rare and serious diseases and protect public health.” About the CSL Centenary Fellowships The Fellowships are competitively selected, high-value grants available to mid-career Australians who wish to continue a career in medical research in Australia. They are open to medical researchers working on discovery or translational research with a focus on rare or serious diseases and are overseen by a selection committee comprising three independent members and two CSL representatives. The 2025 committee was chaired by Dr Andrew Nash. The Fellowships were established to mark 100 years since the establishment of CSL in 1916. Two individual, five-year A$1.25 million fellowships are awarded each calendar year. For further information, visit www.cslfellowships.com.au About CSL CSL (ASX:CSL; USOTC:CSLLY) is a leading global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency, dialysis and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses, CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 30,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSLBehring.com/Vita and follow us on Twitter.com/CSL. For more information about CSL, visit www.CSL.com. # # # Media Contact Name: Kim O’Donohue Mobile: +61 449 884 603 Email: Kim.O’Donohue@csl.com.au