Author Archives: Gareth Macdonald

CGT industry needs skilled staff to fuel further growth says McKinsey

Cell and gene therapy developers need staff with skills according to McKinsey & Company, which says making such therapies is laborious, complex, and expensive and few firms are ready to take on these challenges. The management consulting group wrote about the burgeoning cell and gene therapy industry in a blog post this month, writing that as more cell and gene therapies come to market industry will need to invest in skills. “Five years ago, these treatments were really just ideas…

MilliporeSigma and Agilent aim to extend PAT to real-time release

Merck Life Science (AKA MilliporeSigma) and Agilent Technologies have teamed up to develop process analytical technologies (PAT), citing growing drug industry demand for systems that support real-time release. The collaboration will combine the German drug maker known as MilliporeSigma in North America and the Life Science business of Merck KGaA elsewhere bioprocessing technologies with Agilent’s analytical systems to extend “real-time monitoring and automated process control of critical process parameters (CPPs) and critical quality attributes (CQAs) to downstream processing” the firms…

Multisite manufacturing may not be ideal for cell therapy trials, study says

Cell therapy developers must think carefully before adopting a decentralized manufacturing model say researchers, who suggest the benefits of bedside production are outweighed by technical challenges. The advice, presented in a study in the Journal of Translational Medicine, is based on an assessment of current cell therapy production models which found that the decentralized, multi-production site approach is gaining in popularity, particularly in trials. “For cellular therapy clinical trials involving multiple sites some people are advocating a decentralized model of…

Regeneron to supply Elicio with Libtayo for combo cancer vaccine study

Regeneron has agreed to supply Elicio Therapeutics with the drug Libtayo for clinical trials of its candidate cancer vaccine, ELI-002. The trial will examine the products as a combination in patients with KRAS (Kirsten rat sarcoma virus)-driven tumors including stage III and IV non-small cell lung cancer, stage IV colorectal cancer and unresectable, locally advanced or oligometastatic pancreatic ductal adenocarcinoma. KRAS mutations are among the most prevalent human cancers, driving 32% of lung cancers, 40% of colorectal cancers and 85%…

Standardized handling practices would help cell therapy sector, study

Cell therapy developers need uniform handling, storage, and usage methods say the authors of a new report examining how the market for centrally manufactured products is evolving.  The study – published in the Journal of Translational Medicine in April – looked at the role medical center processing labs play in cell therapy distribution and usage and concluded that such facilities will be a potential bottleneck unless handling processes become more uniform.  “Cell processing laboratories at many academic medical centers are…

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

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…

Gene therapy sector needs tech to meet regulatory requirements

Manufacturing rules are a major challenge for gene therapy firms according to researchers, who say industry needs better tech to meet regulatory requirements. The call for better technology came in a new study published in the journal Molecular Therapy earlier this month, in which progress seen in the cell therapy space was compared with the slower growth of the commercial gene therapy market. “Cell-based therapies have revolutionized the treatment of some blood cancers… Gene therapy, on the other hand, has…

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

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,…

15 year follow-up for gene editing outlined in FDA draft guide

The US FDA has set a 15 year follow-up period for therapeutic gene editing and edited products in new draft guidelines. The guidance – available here – covers therapeutic use of gene editing techniques like ZFN, and CRISPR, as well as gene therapy products made using those techniques. The aim, according to the FDA, is “to assist in the translation of these products from the bench to clinical trials, this guidance includes recommendations for how to assess the safety and…