Author Archives: Gareth Macdonald

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…

Bluebird Bio investigating potential vector role in AML and MDS cases

Bluebird Bio’s suspension of trials of its sickle cell gene therapy LentiGlobin has put the vector used to make it in the spotlight. The US biotech announced the temporary suspension of two LentiGlobin trials – specifically HGB 206 NCT02140554 and HGB 210 NCT04293185 – this week. It said the measure was prompted by reports a patient in group A of the Phase I/II HGB-206 study treated with the therapy five years ago had developed acute myeloid leukemia (AML). Bluebird also…

COVID-19 has put more pressure on vector suppliers, says expert

COVID-19 has increased demand for viral vectors, exacerbating concerns that supply constraints could slow the growth of the cell and gene therapy sector. The ability to insert genetic material into cells using viral vectors is a cornerstone of the modern biopharmaceutical industry. Vectors are used to make everything from cell lines used to produce therapeutic proteins and monoclonal antibodies (mAbs) though to cell and gene therapies. Mismatch As a result, biopharma industry demand for vectors is strong. Indeed, a study…

BMS gearing up to make Breyanzi at Bothell cell therapy plant

Bristol Myers-Squibb (BMS) has set a 24-day production target for Breyanzi and set up a digital platform to let physicians and patients track manufacture of the lymphoma cell therapy in real-time. The US Food and Drug Administration (FDA) cleared Breyanzi (lisocabtagene maraleucel; liso-cel) for the treatment of adults with relapsed or refractory (R/R) large B-cell lymphoma this month. A BMS spokeswoman told us “BMS plans to manufacture Breyanzi for each individual patient at its state-of-the-art cellular immunotherapy manufacturing facilities, initially…

Workforce, tax incentives and US access attract more biotechs to Puerto Rico

CytoImmune Therapeutics and Biosimilar Sciences will invest a combined total of $228 million to set up operations in Puerto Rico. The plan – which was announced by Invest Puerto Rico last week – will see the firms perform novel biologics and cell therapy research and development and hire around 400 employees. A CytoImmune spokesman told us “We chose Puerto Rico due to talented workforce, experienced Pharma service providers, tax incentives, excellent access to US and worldwide shipping, US manufacturing. He…