Therapeutic Class

Biogen to build $200m NC plant to support gene therapy ambitions

Biogen has laid down plans to build a gene therapy manufacturing at its Research Triangle Park (RTP) manufacturing campus in North Carolina. The planned 175,000 square-foot plant – set to create 90 jobs once operational – will support Biogen’s gene therapy pipeline across multiple therapeutic areas. “With this new facility, Biogen is investing in robust and scalable gene therapy manufacturing with the goal of ensuring reliable supply to patients worldwide,” the company said in a statement. “Gene therapy is an…

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…

Ensoma launches to deliver ‘off-the-shelf’ genomic medicine

Ensoma emerges with Series A funding and a collaboration deal with Takeda for its vector-delivered treatments. Ensoma was backed in its Series A financing by 5AM Ventures and Takeda, amongst other investors, with $70 million (€57 million). Takeda’s investment occurs alongside an exclusive worldwide license to develop Ensoma’s ‘Engenious’ vectors for up to five rare disease indications. The agreement includes a potential $100 million investment in upfront and preclinical research payments. Should the candidates progress past the preclinical stage, Ensoma…

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…

Rentschler catapults into the cell and gene therapy space through UK investment

German CDMO Rentschler will set up manufacturing capabilities at the UK’s Cell and Gene Therapy (CGT) Catapult, launching itself into the regenerative medicine space. Family-owned contract development and manufacturing organization (CDMO) Rentschler offers mammalian manufacturing and fill and finish services for biologics but has now expanded into cell and gene therapy production through a deal with the CGT Catapult, based in Stevenage, UK. Rentschler will establish its manufacturing capabilities – including adeno-associated virus (AAV) vector production – through “a significant…

Roche hit for $6.3bn due to biosimilar competition

Roche outlines ‘higher than originally expected’ sales impact from biosimilars worldwide. Roche announced in full year results for 2020 that biosimilars to Herceptin (trastuzumab), Avastin (bevacizumab), and MabThera/Rituxan (rituximab) dented sales by CHF 5.7 billion ($6.34 billion) worldwide. Roche group’s CEO, Severin Schwan, commented in an investor call after the results were revealed that “the impact of biosimilars was significant, somewhat higher than we originally expected.” As a result, sales in the company’s pharmaceuticals division decreased by 8% to CHF…