Therapeutic Class

Polyplus bolsters mRNA portfolio with LNP-alternative reagent

Polyplus has launched an in vivo-jetRNA transfection reagent, which uses a preformed liposome to support and expand its messenger RNA (mRNA) portfolio. Polyplus, a French biotech firm focused on providing biologic and cell and gene therapy research and production, says its in vivo-jetRNA transfection reagent is efficient, stable, and offers a safe delivery system alternative to lipid nano-particle (LNP) delivery. “Non-viral mRNA delivery methods have already proven their efficiency in vaccination through antigen presenting cell modification and in anti-cancer therapy…

Gene therapy firms must consider patients when choosing rAAV production systems

Gene therapy developers need to carefully assess their choice of production platform to make sure it best fits likely demand, clinical timelines, and target patient population size. The advice comes from a study in Current Opinion in Biotechnology that looked at the pros and cons of currently available recombinant adeno-associated viral vector (rAAV) based production systems. The authors compared transient transfection (TT) – which is the most widely used rAAV manufacturing methods – with baculovirus expression vector (BEV) based systems…

Bluebird Skysona approval: ‘A resurgence for HSC gene therapy’

The US approval of bluebird bio’s Skysona (eli-cel) marks a resurgence for the wider hematopoietic stem cell (HSC) gene therapy sector, says AVROBIO. Following the endorsement by an Advisory Committee in June, the US Food and Drug Administration (FDA) granted approval of bluebird bio’s Skysona (elivaldogene autotemcel, or eli-cel) Friday for the treatment of active cerebral adrenoleukodystrophy (CALD). Skysona uses ex vivo transduction with a Lenti-D lentiviral vector (LVV) to add functional copies of the ABCD1 gene into a patient’s…

AbbVie: Manufacturing consistency will affect Humira biosimilar uptake

AbbVie says payors will look to consistent global supply as a key differentiator among the many Humira biosimilar makers poised to enter the US market. AbbVie’s autoimmune monoclonal antibody (mAb) Humira (adalimumab) is the bestselling drug of all time, clocking in over $193 billion for AbbVie (and previously Abbott) since its launch in 2003 to the end of 2021. Last year alone it pulled in $20.7 billion (though lost out to the industry’s top seller spot due to $36.9 billion…

BMS confident Breyanzi capacity issues will be resolved in 2023

Bristol Myers Squibb will apply the same strategy used to increase Abecma supply to increase capacity for Breyanzi as it pushes back projected timelines into next year. Bristol Myers Squibb (BMS) has with its chimeric antigen receptor (CAR) T-cell therapy Breyanzi (liso-cel) “a best-in-class profile,” Christopher Boerner, EVP & chief commercialization officer told stakeholders at the Morgan Stanley Global Healthcare Conference last week. Having been approved in February 2021 for Large B-cell Lymphoma, the therapy won second-line approval for relapsed…

Novel Dual-Plasmid Transfection System Improves Titer And Product Quality In AAV Manufacturing

Adeno-associated virus (AAV) vectors are a widely-used, versatile, and appealing gene therapy delivery platform because of their high safety profile and ability to target many different cell types and transport healthy gene copies into a patient’s cells. The recent clinical successes of AAV-mediated gene therapies have ignited much interest in solutions for the scalable, good manufacturing practice (GMP) production of AAVs, and high demand for outsourced development and manufacturing capabilities. Yet, significant challenges remain, particularly around product quality, productivity, and…

Cali court’s stem cell clinic ruling ‘flawed’ and ‘dangerous,’ says CGT advocacy group

The International Society for Cell & Gene Therapy (ISCT) has slammed the decision of a federal judge in California who ruled surgical procedure using a patients’ stem cells does not fall under the FDA’s regulatory jurisdiction. In 2017, the US Food and Drug Administration (FDA) took a series of actions “to remove unproven, potentially harmful treatment used in ‘stem cell’ centers targeting vulnerable patients.” This included the issuing of Form 483s and warning letters, the seizing of vials of materials…

Takeda and Cryoport team to standardize cell therapy supply chain

A standardized approach to collecting, processing, and storing cell therapy starting materials can reduce variations in manufacturing outcomes and simplify supply chain management, says Cryoport’s CEO. Temperature-controlled supply chain services firm Cryoport has partnered with Takeda’s plasma donation and collection business BioLife Plasma Services to offer standardized cell therapy collection, processing, and storage services for cell therapy products across the US and Europe. The collaboration, of which no financial details have been divulged, aims to establish an integrated GMP-compliant supply…

Orca Bio taking inhouse approach to cell therapy with Sacramento plant

“It’s important we maintain control over the process to ensure we are meeting the highest standards of quality,” says cell therapy developer Orca as it announces a 100,000 square-foot plant. Orca Bio’s lead product, the cell therapy Orca-T, is in Phase III clinical trials for several of the most prevalent blood cancers. As such, the firm is looking beyond its clinical manufacturing site in Sacramento, California and is constructing a 100,000 square-foot commercial facility nearby with the capacity to manufacture…

Neukio Bio raises $50m for allogenic CAR-T therapies

Shanghai, China-based Neukio Biotherapeutics has completed a $50 million Series A-1 funding to support development of its cell therapy molecules, especially off-the-shelf allogenic CAR-T therapies. After raising $40 million in an Angel round last year, the company will use these latest funds to accelerate preclinical/clinical validation of induced pluripotent stem cell (iPSC)-derived off-the-shelf CAR-NK cell therapy products, while also expanding its team and operations. This latest funding was led by CD Capital, with participation from Alwin Capital and Surplus Capital…