Author Archives: Gareth Macdonald

Single-use techs are garbage, eventually, and that’s great for biopharma

Biopharma is starting to use single-use technologies more widely but understanding the pros and cons is vital say experts. The key characteristic of any single-use technology is its disposability. Being able to throw away rather than clean equipment after use is a major benefit. Cleaning a stainless steel system takes time and money says Andrew Sinclair, founder of Biopharm Services. “Cleaning a typical stainless steel system can be complex as the equipment and associated pipework needs to be cleaned together…

Zolgensma: We all know the price, but how is it made?

Making Zolgensma, the new “world’s most expensive drug,” is a month-long process that depends on vector tech licensed from Regenxbio. Zolgensma is a gene therapy. It is designed to halt progressive spinal muscular atrophy (SMA) – motor neurone loss and muscle wasting – in people who have a defective version of the gene SMN1. The drug addresses the underlying causes of SMA according to Novartis spokesman Eric Althoff. He told us, “Zolgensma provides a functional copy of the human SMN…

Costs and quality will shape cell and gene therapy tech development

Cell and gene therapies as well as wider industry desire for cost efficiency will shape bioprocessing tech innovation, say experts. Biopharma interest in cell and gene therapies is increasing. Products like Kymriah, Luxturna and Yescarta are the tip of an R&D iceberg according to PhRMA, which says around 300 such treatments are in development. The trend has been well received, with various groups suggesting it will yield treatments for untreatable conditions (see here, here and here, for examples). Investors have…

Making rare disease successes more common

Complex pathologies and small patient populations make rare disease R&D difficult according to Alexion, which says recent FDA guidelines are welcome. At large biopharma firms, rare disease drug R&D projects are becoming common. Their high revenue potential combined with IP advantages afforded to such products makes them an attractive focus for developers. For example, orphan drug designation entitles firms to reduced fees, tax breaks for trials and extended market exclusivity. Investors also welcome such designation. According to one study, developers…

Antibody–Drug Conjugate News: From BioProcess Insider

The following news items have appeared on the BioProcess Insider site over the past year. Together they indicate the direction in which the ADC sector is moving. BEYOND ADCETRIS — SEATTLE GENETICS AIMS FOR BIG BIOPHARMA STATUS 30 April 2018: Seattle Genetics is building toward a bigger future. For the first quarter 2018, the company’s total revenues grew to US$141 million (€116 million) compared with $109 million in the same period last year. This was attributed to a 36% increase…

Breathing space: Inhalable biologics interest prompts Intertek expansion

Interest in inhaled biologics is increasing says Intertek, which has announced plans to expand its testing facility in Melbourn, near Cambridge. Intertek will double capacity with a new 20,000 square-foot lab kitted out with technologies use to assess inhaled and nasal formulations of large molecule drugs. It also plans to hire 100 employees over the next four years. “As companies are increasingly looking at biologics for non-invasive delivery platforms like inhaled and nasal, there is expected to be an increased…

FDA pledges to support cell and gene therapy manufacturing innovation

Manufacturers aiming to follow Yescarta and Kymriah to market can count on FDA support after the agency said it will help cell and gene therapy firms adopt better production methods. A US Food and Drug Administration (FDA) spokesman told Bioprocess Insider the agency’s emerging technology team has been tasked with working with cell and gene therapy firms to “identify innovative approaches for efficient product manufacturing.” The move is part of a wider FDA effort to support modernization of drug manufacturing…

PureTech cites Big Pharma tech partnerships as 2018 highlights

PureTech Health says delivery technology deal with Roche was key part of its financial performance in 2018. The US biotech made the comments last week. It explained that its revenue growth for the year – $20.7 million (€18.5 million), up from $2.5 million in 2017 – was due in part to the Roche accord inked last July. CEO Daphne Zohar also predicted the deal will shape future performance. “We are now engaged in collaborative research with Roche to advance our…

Lonza goes live with microbiome CMO joint venture

Lonza says GMP know-how and drug delivery tech are core to its live biotherapeutics joint-venture formed with Chr. Hansen. The Swiss contractor and Danish bioscience firm set up the contract manufacturing organization (CMO) in April, citing biopharma interest as the driver. Live biotherapeutic products (LBPs), as the name suggests, are living organisms. Unlike probiotics used to try to improve general health, LBPs are drugs intended to treat or cure disease. “LBPs are regulated as drugs and have to go through…

GeoVax targets WHO goal with multi antigen malaria vaccine

GeoVax Labs says the malaria vaccine it is developing with Leidos could achieve efficacy levels called for by the WHO. US firms GeoVax and Leidos agreed to develop a P. falciparum vaccine in March, expanding on an R&D partnership they formed last November. Under the deal, GeoVax will make a vaccine able to deliver DNA coding for parasite proteins. Cells ‘infected’ by the vaccine will express the proteins, provoking an immune response. Leidos spokeswoman Erin Tindell told us the DNA…