Cell Therapies

How Are Service Companies Responding to the Increased Interest in New Modalities, and What Regulatory and Market Hurdles Still Exist?

On Tuesday, April 30, 2019, Dan Stanton moderated a CPhI Theater roundtable discussion about the ways that biomanufacturers are confronting great demand for monoclonal antibody (MAb) therapies, antibody drug conjugates (ADCs), and other emerging treatments that depend on complex chemistries. Featuring Juan Cordova (Principal Scientist of Bioconjugation at Abzena), Dawn Ecker (a consultant with the BioProcess Technology Group at BDO), and Scott Miller (Senior Scientific Advisor at Carbogen Amcis AG), this panel explored current challenges to upstream processing for antibody…

Immunotherapy: Taking Aim at Solid Cancers

As cell and gene therapies arrive on the market, all eyes have focused on autologous chimeric antigen receptor (CAR) T – cell therapies. At the 2019 Phacilitate Leaders World and Stem Cell Summit in Miami, FL, delegates looked at where the biopharmaceutical industry is going in the cellular immunotherapy space. Whether for off-the-shelf CAR T-cell products, personalized cancer vaccines, or modified natural killer (NK) cells derived from human induced pluripotent stem cells (iPSCs) — cell and gene therapy development is…

Supply Chain Solutions for Cell and Gene Therapy Companies

Stakeholders across the supply chain stress that quality of starting material will be key to the success of cell and gene therapies. This is a topic that has created issues in the past, is puzzling the industry presently, and is likely to cause more problems going forward. This topic was front and center at the 2019 Phacilitate Leaders World and World Stem Cell Summit in Miami FL, with presentations focusing on supply chain solutions to address these complex challenges; cell…

Automation in Cell and Gene Therapy Development

The US approvals of chimeric antigen receptor (CAR) T-Cell therapies Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) and gene therapy Luxturna (voretigene neparvovec) in 2017 heralded a “new frontier of medicine.” But with great innovation comes great costs and criticism (such as the Kymriah’s US$475,000 price tag). Many companies argue that these one-off therapies represent good value for patients and payers compared with traditional treatments, however, no matter your perspective, the COGs picture for cell and gene therapies isn’t good and…

Manufacturing of Cell and Gene Therapies

The peak of demand for curative cell and gene therapies will be unlike that of traditional drugs and thus could cause forecasting and overcapacity issues going forward. Predicting the future is always difficult, and poor decisions can be costly and highly damaging for a company. At the 2019 Phacilitate Leaders World and Stem Cell Summit in Miami, FL, several presentations and conversations focused on the forecasting dilemma and how manufacturing needs innovation, just to name two. This eBook details these…

eBook: Allogeneic Cell Therapies Commercialization Strategies

Manufacturers of allogeneic cell therapies face development and commercialization challenges unlike those of traditional cell therapies. In a discussion with Phil Vanek of GE Healthcare, we outline several of the key challenges of processing these products and bringing them to market. Approaches for reducing development costs and lowering pricing are highlighted, and a separate analysis presents three pricing models specific for allogeneic “off the shelf” cell products.

The Role of Single-Use Polymeric Solutions in Enabling Cell and Gene Therapy Production – Part 3: Best Practices for Supplier Selection, Qualification, and Validation to Ensure Supply Chain Security

Bio-Process Systems Alliance (BPSA) was formed in 2005 as an industry-led international industry association dedicated to encouraging and accelerating the adoption of single-use manufacturing technologies used in the production of biopharmaceuticals and vaccines. Corporate members include plastic equipment suppliers, service providers, and users in the biopharmaceutical industry who share this mission. A key focus of BPSA’s core activities is to educate its members and others through sharing of information and development of best practice guides that help suppliers, users, and…

The Role of Single-Use Polymeric Solutions in Enabling Cell and Gene Therapy Production –
Part 2: Regulatory Overview

The Bio-Process Systems Alliance (BPSA) was formed in 2005 as an industry-led international industry association dedicated to encouraging and accelerating the adoption of single-use manufacturing technologies used in the production of biopharmaceuticals and vaccines. Corporate members include plasticequipment suppliers, service providers, and users in the biopharmaceutical industry who share this mission. A key focus of BPSA’s core activities is to educate its members and others through sharing of information and development of best practice guides that help suppliers, users, and…

The Role of Single-Use Polymeric Solutions in Enabling Cell and Gene Therapy Production: Part 1: Introduction and Manufacturing Process

by Bio-Process Systems Alliance Cell and Gene Therapy Committee The Bio-Process Systems Alliance (BPSA) was formed in 2005 as an industry-led international industry association dedicated to encouraging and accelerating the adoption of single-use manufacturing technologies used in the production of biopharmaceuticals and vaccines. Corporate members include plastic-equipment suppliers, service providers, and users in the biopharmaceutical industry who share this mission. A key focus of BPSA’s core activities is to educate its members and others through sharing of information and development…

A Perspective on GMPs for Cellular Therapy Commercialization

Cellular therapies can be classified by therapeutic indication, by cell types, and by whether cells are taken from and administered to the same individual (autologous) or derived from healthy donors (allogeneic). Regulatory classification of cellular therapies differentiates among minimally manipulated cells for homologous use, transplants or transfusions, and cells that are more than minimally manipulated and regulated as medicines. Medical cellular therapies must meet quality, safety, and efficacy standards to obtain marketing authorization (1–8). Such therapies can be subdivided into…