Therapeutic Class

Limula on the future of cell therapies: Closed, automated, decentralized

An in-situ centrifugation-based point-of-care system could help push industry towards the decentralized and automated model needed to make cell therapies affordable and reliable, according to Limula Biotech. The approval of Novartis’ Kymriah (tisagenlecleucel) in 2017 was a milestone in the advanced therapy space, marking the arrival of the first genetically-modified autologous T-cell immunotherapy to the market. Since then, a handful of other autologous cell therapies have received the regulatory thumbs up – most recently Bristol-Meyer Squibb’s Breyanzi (liso-cel) and Abecma…

CBM and UPenn enter gene therapy manufacturing pact

A five-year deal will see the Center for Breakthrough Medicines (CBM) gain commercial rights to certain gene therapy manufacturing and analytics platforms from the University of Pennsylvania. The King of Prussia, Pennsylvania-based contract development and manufacturing organization (CDMO) has entered a collaboration with the University of Pennsylvania Gene Therapy Program (GTP) to help small biotech firms, universities, and large pharma firms advance gene therapies through the clinic. The collaboration brings CBM access to UPenn’s gene therapy expertise, which has helped…

Lonza teams with Agilent to make cell therapy Cocoon platform ‘smarter’

Lonza will work with Agilent to define the ideal Critical Quality Attributes (CQAs) for cell therapy manufacturing and help better control the in-process controls and analytics of its patient-scale Cocoon platform. Lonza’s Cocoon platform is a single system that can be used for a variety of different autologous cell therapy protocols, with each patient batch produced in a single disposable cassette customized to their specific process. The automated point-of-care manufatcuring system was developed by Octane Biotech, and though Lonza has…

With acquisitions integrated, Curia unveils mRNA offering

CDMO Curia is leveraging recent acquisitions to offer end-to-end messenger RNA (mRNA) services, saying COVID-19 vaccines essentially served as proof of concept for the new modality. 2021 was an eventful year for contract development and manufacturing organization (CDMO) Curia. Along with a rebrand from its AMRI heritage, the company acquired LakePharma and Integrity Bio and – having integrated the entities into its business – has now launched a full mRNA service for its customers. “Curia has combined more than 30…

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. “In its notice to Sangamo, Sanofi indicated that its termination relates…

Amgen: Not treating biosimilars as ‘stepchild’ is key to success

Amgen says using the same development expertise and manufacturing capabilities for both its biosimilar and novel biologic programs sets it apart in the industry. Pharma firm Amgen is one of a handful of firms that has successfully invested in both innovator products and biosimilars – biologics that are very similar to reference biologics and for which there are no clinically meaningful differences in terms of safety, purity, and potency. With expected 2021 biosimilar revenues set to reach $2 billion, CEO…

New year, new gene therapy plants from Pfizer and Expression

Christmas came early for Pfizer and Expression Therapeutics, which cut the ribbons on viral vector manufacturing plants in North Carolina and Ohio, respectively. Pfizer’s gene therapy division celebrated the new year with an expanded manufacturing footprint, having opened the first of three facilities at its site in Durham, North Carolina on December 15. The 85,500 square-foot plant will support clinical manufacturing of Pfizer’s gene therapy pipeline and represents an investment of $68.5 million by the firm. The facility will create…

Novartis to boost gene therapy pipeline through $800m Gyroscope buy

Novartis will add a gene therapy candidate in Phase II trials for an advanced form of dry age-related macular degeneration through the addition of Gyroscope Therapeutics. Novartis will gain GT005, an Adeno-Associated Virus-2 (AAV)-based gene therapy candidate intended to be a one-time treatment for geographic atrophy (GA) – an advanced form of dry age-related macular degeneration (AMD) that leads to progressive and irreversible vision loss – through the proposed acquisition. Novartis is one of the leaders in the gene therapy…

RNA sector set for progress thanks to decades long research drive

The RNA drug sector has overcome some significant hurdles according to Alynlam Therapeutics CEO John Maraganore, who says industry efforts to work with these challenging molecules have laid the foundations for growth. “The scientific obstacles are best defined here, small interfering RNA molecules are large. They’re charged. They’re susceptible to biological degradation. They’re also prone to activated immune sensor systems. And so the challenge of making drugs out of small interfering RNAs was really a formidable scientific challenge that needed…

BrainStorm’s cell therapy: Catalent high on NurOwn supply

BrainStorm Cell Therapeutics is eying the potential launch of NurOwn and has selected CDMO Catalent to produce the autologous cell therapy from its site in Texas. Known as NurOwn, MSC-NTF is cell therapy candidate being investigated in Phase III trials for Amyotrophic Lateral Sclerosis (ALS) by BrainStorm. The potential therapy is made from bone marrow-derived mesenchymal stem cells (MSCs) taken from the patient, which are expanded and differentiated ex vivo before being converted into MSC-NTF cells that secrete high levels…