Therapeutic Class

Novo Nordisk and bluebird to develop ‘once and done’ hemophilia gene therapy

The three-year collaboration will use bluebird bio’s mRNA-based megaTAL gene editing technology to develop a one-off therapy for patients with hemophilia. Novo Nordisk has had a longstanding focus on the hemophilia space. The Danish drugmaker has marketed several replacement factors for hemophilia patients and has a monoclonal antibody in Phase II studies: Concizumab, a Tissue Factor Pathway Inhibitor (TFPI) intended for bleeding prevention after subcutaneous administration. But a partnership announced this week with regenerative medicine firm bluebird bio will look…

Novartis supply-ready for approved wet AMD antibody fragment

Novartis says it is confident in its ability to supply Beovu (brolucizumab) after the single-chain antibody fragment received US FDA approval for treating wet AMD. The US Food and Drug Administration (FDA) this week approved Novartis’ anti-VEGF product Beovu for the treatment of patients with wet age-related macular degeneration (AMD) on a three-month dosing interval. A Novartis spokesperson did not divulge where the antibody fragment is made but told this publication the firm is confident in its ability to meet…

‘Unprecedented’ HPV vaccine demand to fuel more Merck capacity investments

Having recently invested over $1.6 billion in its network, Merck says it is committed to further expand the supply of its HPV vaccines as worldwide demand continues to spiral. Speaking at the at Morgan Stanley 17th Annual Global Healthcare Conference last month, Roger Perlmutter, EVP and president of Merck Research Laboratories, said human papillomavirus (HPV) vaccines Gardasil and Gardasil 9 were the products that have the dominant near-term commercial potential for his firm. But a lack of manufacturing capacity at…

Moderna to expand mRNA plant with tech development center

Moderna will build an 85,000 square-foot technical development center at its recently opened manufacturing site in Massachusetts to support its messenger RNA (mRNA) pipeline. mRNA products themselves are neither small molecules nor traditional biologics, but rather a ‘set of instructions’ directing cells to, essentially, ‘use the human body as the bioreactor’ and make the proteins needed to prevent or fight diseases. Moderna Therapeutics has over 20 mRNA-based products in its pipeline, including prophylactic vaccines, cancer vaccines, intratumoral immuno-oncology candidates, and…

Vector market moving in right direction: Shortage driving CDMO M&A

Demand for viral vectors has emerged as a major M&A driver in the gene therapy CDMO space says an industry expert. Viral vectors are hollow viruses developers can fill with genetic material. When one of these vectors ‘infects’ a cell the genes inside are inserted and expressed as proteins. Vectors are vital for the production of gene therapies, including new products like Spark Therapeutics’ Luxturna (voretigene neparvovec-rzy) and AveXis’ Zolgensma (onasemnogene abeparvovec-xioi). The problem is that, for the past few…

In the pipeline: Surge of cell and gene therapies likely in 2020

Over 20 cell and gene-based therapies are expected to be filed or to receive approval decisions over the next 18 months, according to the Alliance for Regenerative Medicine (ARM). Earlier this year, the US Food and Drug Administration (FDA) laid out its action plan to deal with the large upswing in the number of investigational new drug (IND) applications for cell and gene therapies it is receiving. “The FDA commissioner has anticipated that he thinks there will be between 10-20…

Yescarta 2.0? How Kite plans to scale out CAR-T therapies

CAR-T pioneer Kite Pharma says scaling out capabilities, embracing digital technologies, and industry collaboration are among the efforts needed to bring cell therapies to maturity. In 2017, Gilead company Kite Pharma became the second company to achieve US regulatory success for a chimeric antigen receptor (CAR) T-cell therapy. Two years on, and while sales of the product Yescarta (axicabtagene ciloleucel) are rising – Q2 2019 saw a 25% quarterly growth to $120 million –the sector itself has somewhat stalled with…

Treefrog wins EU funding for mass-produced cell therapy tech

Treefrog Therapeutics has received €10 million ($11 million) over the past five months to support its mass-produced pluripotent stem cell platform C-Stem. In September, Bordeaux, France-based Treefrog Therapeutics received €450,000 from the French government and €2.4 million from the EU’s Horizon 2020 program to help acelerate the cGMP certification and scale-up of its C-Stem technology platform. It comes five months after the firm raised €7 million in a Series A funding. “C-Stem is a technology that enables the mass-production of…

Zolgensma data: ‘Heavy-handiness’ of FDA a warning to industry, not just Novartis

The US FDA may have made an example of Novartis in the way it handled the revelation of Zolgensma data manipulation, says the Alliance for Regenerative Medicine. A month after the US Food and Drug Administration (FDA) approved the one-time gene therapy Zolgensma (onasemnogene abeparvovec), marketing authorization holder Novartis alerted the agency to a data manipulation issue that impacted the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA). In its response,…

GSK pays Immutep on advancement of Ab for Ulcerative Colitis

The first patients have been dosed in a Phase II trial for GSK2831781, GSK’s ulcerative colitis candidate based on Immutep’s depleting anti-LAG antibody technology. In 2010, GlaxoSmithKline (GSK) licensed ImmuTune-IMP731, a therapeutic antibody for the treatment of autoimmune diseases, from Immutep in a deal that included milestone payments of £64 million ($80 million). This week, GSK is paying out £4 million of this due to the first patient being dosed in GSK’s Phase II clinical trial evaluating the candidate –…