Author Archives: Gareth Macdonald

Lifecycle approach could cut cell and gene therapy costs

Cell and gene therapy firms should consider a ‘lifecycle approach’ to process development and manufacturing to reduce cost of goods, says ElevateBio. Developing cell and gene therapies is highly complex. Such work requires specialist skills and hard to make materials like vectors, the lack of capacity for which has been raised as a constraint in the scientific and mainstream press. But cell and gene therapy firms also need to keep commercialization in mind according to Mike Paglia, COO at cell…

BARDA to give Emergent another $23m for tech after J&J jab mix-up

BARDA to give Emergent Biosolutions an extra $23 million for new tech after plant mix up ruined millions of doses of J&J’s single-shot COVID vaccine. The facility in question is Emergent’s plant in Baltimore, Maryland which made the news after it emerged the contract development and manufacturing organization (CDMO) had mistakenly mixed ingredients of the J&J jab with those of the AstraZeneca shot, rendering 15 million doses unusable. The incident prompted the Biden administration to place Johnson & Johnson in…

Biotech and CDMOs need to evolve to support innovate therapies, says Ilya Pharma

CDMOs and the cell and gene therapy sector need to evolve to support the increasing number of innovative therapies entering trials, says Ilya Pharma. Ilya CEO Evelina Vågesjö made the comments about innovative therapy manufacturing after the Sweden-based biotech released first-in-human (FIH) trial data indicating ILP100 – a bacteria-based therapeutic candidate designed to treat chronic wounds – shortens healing time by six days. “We have evaluated multiple CMOs [contract manufacturing organizations] for clinical material production and commercial supply already and…

CDMO and COVID delays push back Vaxcyte’ pneumococcal vaccine IND

Vaxcyte will file VAX-24 pneumococcal conjugate vaccine later than planned citing capacity constraints at CDMO Lonza and the COVID-19 pandemic. The California biotech had intended to file the vaccine for US FDA review early this year, however, according to its Q4 results presentation, submission is now planned during the first half of 2022. Vaxcyte said it expects to submit an IND application “between January and June 2022 and plans to announce top-line data from the ensuing Phase I/II clinical study…

BIO: Sputnik V should be judged on same data as other COVID jabs

Russia’s Sputnik V COVID-19 vaccine should be judged by the same safety and efficacy standards applied to jabs made by Moderna, Pfizer, AstraZeneca, and J&J according to industry group BIO. The Amsterdam-based EMA began reviewing the vaccine – also called Gam-COVID-Vac – last week after R-Pharm Germany GmbH submitted an approval application. A spokeswoman told us “The CHMP’s decision to start the rolling review of the Sputnik V COVID-19 vaccine is based on results from laboratory studies and clinical studies…

Busulfan not likely cause of Bluebird Bio trial SAEs says CRISPR Therapeutics

CRISPR Therapeutics says the conditioning agent busulfan is unlikely to be the cause of adverse events that prompted Bluebird Bio to halt LentiGlobin trials. As a medicine busulfan is used to treat chronic myelogenous leukemia. It works by slowing or stopping the growth of cancerous cells. It is also used in cell and gene therapy as a conditioning agent to destroy bone marrow to prepare patients for transplant. Busulfan hit the headlines this month after Bluebird Bio started investigating it as…

Precigen: ‘Electroporation a game changer for cell therapy production’

Precigen says the electroporation device used to make its UltraCAR-T therapies is a game changer for cell therapy production. The US biotech spoke about the technology – the UltraPorator – during its recent Q4 call, citing US FDA approval for its use in the production of investigational UltraCAR-T therapies as a highlight of 2020. According to CEO Helen Sabzevari, “UltraPorator is a potential game-changer for manufacturing of personalized UltraCAR-T therapies as it significantly reduces processing time and contamination risk. “While…

Busulfan role in Bluebird’s woes could impact gene therapy sector

Cell and gene therapy developers will have a close eye on cancer cases linked to Bluebird Bio’s LentiGlobin, say analysts. Earlier this month Bluebird halted two trials of LentiGlobin in sickle cell disease – specifically HGB 206 NCT02140554 and HGB 210 NCT04293185. It said the decision was prompted by a case of acute myeloid leukemia (AML) in a patient in group A of the Phase I/II HGB-206 study and a case of myelodysplastic syndrome (MDS) in a patient in Group C of the…

COVID-19 MAbs should be made with combination in mind says US FDA

Monoclonal antibody (mAb) manufacturers aiming for an EUA should prepare for their SARS-CoV-2 therapies to be combined with others, according to the US FDA. The US regulator issued the advice in guidance this week in which it also urged developers to collaborate and share data. The FDA said, “Strongly recommends that individual monoclonal antibody products be developed with the expectation that they will be combined with one or more monoclonal antibody products that bind to different epitopes to minimize the…

Novo Nordisk and Vertex update on rival diabetes cell therapy efforts

Novo Nordisk plans to file an IND for cell therapy for type 1 diabetes in the next few years, setting the stage for a battle with Vertex which aims to start Phase I/II trials of its candidate this year. Denmark’s Novo Nordisk shared details of its stem cell-based candidate during its Q4 call recently, explaining it will submit the therapy along with several investigation new drug (IND) applications over the next few years. CSO Mads Thomsen told analysts “The first…