Therapeutic Class

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…

Partnerships abet vendors in tackling cell & gene logistics and automation

With Thermo Fisher teaming with automated tech company Scinogy and GE Healthcare collaborating with logistics firm World Courier, both vendors have increased their advanced therapy services. The recent approval of Novartis’ one-off gene therapy Zolgensma (onasemnogene abeparvovec) has cemented the advent of advanced therapies. With hundreds more cell and gene therapies in development, the US FDA has predicted that up to 20 such products will be approved each year by 2025. Vendors, therefore, have been increasing their commercial services technologies…

Zolgensma approval marks success for REGENXBIO’s vector platform

REGENXBIO could receive up to $80 million after the US FDA approved Novartis/AveXis’ gene therapy Zolgensma (onasemnogene abeparvovec), which uses the firm’s NAV Technology Platform. Last week, the US Food and Drug Administration (FDA) approved Zolgensma, a single-dose gene therapy developed by Novartis acquisition AveXis, for the treatment of children less than two years of age with spinal muscular atrophy (SMA). The approval triggered a $3.5 million (€3.1 million) milestone payment to Maryland-based biotech REGENXBIO. Zolgensma is based on REGENXBIO’s…

Personalized medicines need personalized pricing plans, Orchard

There is no one-size-fits-all model for cell and gene therapy pricing plans says Orchard Therapeutics, but industry must adapt a system set up for chronic care to incorporate curative one-off treatments. There have been questions over how payors and insurance firms would cope with such next-generation therapeutics have been asked ever since Novartis launched its first cell and gene therapy product Kymriah (tisagenlecleucel) in 2017 at a list price of $475,000. But the recent US Food and Drug Administration (FDA)…

Novartis wins approval for $2.1m gene therapy Zolgensma

Novartis says it is ready to launch Zolgensma (onasemnogene abeparvovec) within the next few weeks after receiving FDA approval for its spinal muscular atrophy (SMA) single-dose gene therapy. Zolgensma, a single-dose, one-time gene therapy has been approved to treat children less than two years of age with spinal muscular atrophy (SMA). “Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” acting US Food and Drug Administration (FDA) commissioner…

FDA issues draft guidance on biosimilar analytical studies

The draft guidance advises industry to minimize differences between a biosimilar and its reference biologic including in the choice of expression system and manufacturing process. The US Food and Drug Administration (FDA) has been active in encouraging biosimilar development of late, publishing its long-awaited interchangeability guidance earlier this month. In its latest push the Agency has issued draft guidance entitled: ‘Development of Therapeutic Protein Biosimilars: Comparative Analytical Assessment and Other Quality-Related Considerations Guidance for Industry.’ The document makes recommendations on…

Off-the-shelf CAR-T a ‘gamechanger’ for multiple myeloma, says Allogene

Allogene expects manufacturing specifications to be a focus of IND reviews as it looks to bring an off-the-shelf CAR-T therapy for multiple myeloma through the clinic. Chimeric antigen receptor (CAR) T-cell therapies have been described as “one of the newest, emerging weapons in the fight” against multiple myeloma, a cancer affecting around 300,000 Americans every year. Some of the candidates in the clinic for multiple myeloma include Bluebird and Celgene’s BB21217, Celgene’s JCARH125 and Janssen/Legend Biotech’s LCAR-B38M, but these –…

Cryoport acquires Cryogene for $20.5m, adds Texas biostorage site

The combination of cold-chain logistics and biostorage places Cryoport in good stead for the arrival of allogeneic cell therapies, an analyst says. Cryogene’s proximity to Lonza’s Houston site is also a boon. Cryoport, a provider of temperature-controlled logistics solutions specifically in the cell and gene therapy space, paid $20.5 million (€18.3 million) in cash for Houston, Texas-based biostorage and biobanking firm Cryogene. Cryogene brings a 21,000 square-foot biostorage facility – which specializes storing biological specimens, materials and samples – to…

ElevateBio proposes centralized model for cell & gene startups

Neither a CDMO nor an end-user, ElevateBio is looking to change the paradigm in cell and gene therapy development through it’s a centralized R&D and manufacturing model. Recently launched through a $150 million (€134 million) Series A financing, ElevateBio intends to create and grow a portfolio of cell and gene therapy companies using shared expertise and facilities through its R&D, process development and cGMP manufacturing subsidiary Basecamp. “BaseCamp is not a CDMO [contract development and manufacturing organization],” ElevateBio’s CEO David…