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Gamida’s off-the-shelf lymphoma cell therapy set for trials after hold lifted

The US FDA has released Gamida Cell from a clinical hold imposed last year, clearing the way for trials of GDA-201, a candidate off-the-shelf treatment for large B cell lymphomas. The Boston biotech firm announced the decision last week, also revealing that the US regulatory agency cleared its investigational new drug (IND) application for the product. CEO Julian Adams said, “FDA clearance of our IND for the cryopreserved formulation of GDA-201 represents a significant milestone for the company and reflects…

ATMPS gets US patent for blockchain for personalized meds

by Gareth Macdonald

US authorities have granted ATMPS Ltd a patent for the use of blockchain technologies in the delivery of personalized medicines. ATMPS’ technology – known as Hataali – is designed to track personalized medicines from “vein to vein,” in other words from the initial harvesting of cells from a clinical trial participant to the production facility and back through a series of nodes. CEO Raja Sharif told us “Hataali is an incredibly flexible platform with a large array of potential uses…

Novartis CGTs first quarter 2022: Zolgensma up, Kymriah down

by Dan Stanton

Novartis reports increased sales for gene therapy Zolgensma due to expanded global access, but the failure in the second-line DLBCL setting will affect Kymriah sales. For Swiss Pharma giant Novartis’ cell and gene therapy (CGT) division, first quarter 2022 results were mixed. On the positive side, Zolgensma (onasemnogene abeparvovec) pulled in $363 million during the quarter, up 18% year-on-year due to increased access outside of the US. Sales of the one-time spinal muscular atrophy (SMA) gene therapy — approved in the…

Cash strapped Bluebird cuts staff and focuses on late stage gene therapies

by Gareth Macdonald

Bluebird Bio is cutting its workforce and prioritizing late phase gene therapy projects to reduce costs. The US biotech announced the staff cuts this week, explaining the aim is to deliver up to $160 million in operational savings over the next two years. It did not share further details. At the start of the year, Massachusetts-based Bluebird said it employed 518 people, 330 of whom work in R&D with 188 engaged in commercial, finance, legal, business development, human resources, information technology,…

Buoyant ADC market drives Sterling expansion

by Dan Stanton

A year after acquiring ADC Bio, Sterling Pharma is expanding its Deeside, UK facility to better serve the growing antibody-drug conjugate (ADC) market. Sterling entered the ADC space in April 2021 through the acquisition of ADC Bio. The deal brought with it a 6,500 square-meter site in Deeside, Wales housing ADC and bioconjugation development laboratories. A year on and Sterling is committing a further £1 million ($1.3 million) to the site, increasing laboratory space from 275 to 419 square meters,…

Novartis’s $115m pays off as FDA approves second Zolgensma site

by Dan Stanton

The Novartis facility in North Carolina will begin shipping Zolgensma gene therapy doses immediately after receiving US FDA commercial licensure approval. The Swiss Big Pharma firm announced yesterday the US Food and Drug Administration (FDA) has granted approval to its multi-product gene therapy manufacturing facility in Durham, North Carolina. The 170,000 square-foot facility site will primarily be used to produce Zolgensma (onasemnogene abeparvovec. Novartis added Zolgensma to its pipeline through the $8.7 billion acquisition of AveXis in May 2018 and…

Bayer on keeping CGT acquisitions at arm’s length

by Dan Stanton

Keeping biotech bolt-ons separate from its main organization is essential in retaining agility, speed, and innovation for cell and gene therapies, says Bayer’s Jens Vogel. Despite relatively few reaching commercialization, cell and gene therapies (CGTs) have become a solid part of the wider pharmaceutical landscape with numerous big firms dabbling in the sector. German biopharma giant Bayer became a serious fixture in the advanced therapy space in 2019 when it bought out cell therapy developer BlueRock Therapeutics. The following year,…

Latent application: Moderna lauds mRNA manufacturing for herpes, HIV and beyond

by Dan Stanton

Moderna says the flexibility and footprint of mRNA production can overcome scaling-up issues seen with current latent virus vaccines like GSK’s Shingrix. Moderna entered the ‘Big Pharma’ space in 2021 when its COVID-19 vaccine Spikevax became the second messenger RNA (mRNA) product to achieve commercial success. Last year, 807 million doses of the vaccine were sold, bringing in $17.7 billion for Moderna. For comparison, Moderna reported total sales in 2020 of $803 million and $60 million in 2019. Going forward,…

Sanofi refills ADC pipeline with Seagen pact

by Dan Stanton

The deal will see Sanofi and Seagen collaborate on antibody-drug conjugate (ADC) candidates targeting up to three cancer targets. Sanofi will leverage its monoclonal antibody (mAb) technology with Seagen’s ADC platform in the partnership. Specific financials are scarce, but Sanofi will make an undisclosed payment to Seagen for each of the three targets as they are selected. The development costs and future profits will be shared between the two companies. “This collaboration will enable the synergistic combination of molecules and platforms to produce candidate medicines with the potential of bringing renewed hope to cancer patients…

J&J taking ‘disciplined’ approach to CAR-T rollout

by Dan Stanton

“CAR-T cell manufacturing is not a finished product,” says J&J EVP Mathai Mammen as he speaks about his firm’s rollout of recently approved myeloma therapy Carvykti. Last month, Johnson & Johnson received the regulatory thumbs up for its chimeric antigen receptor T-cell (CAR-T) therapy Carvykti ( ciltacabtagene autoleucel; ciltacel), developed with China-focused partner Legend Biotech. The therapy is the second BCMA CAR-T approval – Bristol-Myer Squibb’s Abecma (ide-cel) received FDA approval in March 2021 – and the first cell therapy success…