U.K.-based medical charity LifeArc, and their partners, the Medical Research Council (MRC) and the Biotechnology and Biological Sciences Research Council (BBSRC), announced plans for investing $24 million (£18 million) to create cutting-edge gene therapy innovation hubs.
The hubs—at King’s College London, NHS Blood and Transplant in Bristol, and the University of Sheffield—will provide GMP-grade manufacturing services, including viral vector production, to gene therapy developers from industry and academia.
Catriona Crombie, PhD, associate director, technology transfer, and head of LifeArc’s philanthropic fund, says the aim is to address the challenge of moving gene therapies out of the academic space and into the commercial environment.
“Priority will be given to academic led projects but where there is capacity, industry can apply to have their products made at the hubs. There will be a coordinating committee who will have responsibility for assessing and allocating projects across the network,” she tells GEN.
“The coordinating Committee will oversee the creation and ongoing operation of the Hub network to promote sharing of knowledge and capabilities, engage with the academic community, and foster interactions with commercial organizations to facilitate the onward development of new genetic medicines.”
Academic developers, in particular, stand to benefit from manufacturing innovation and extra capacity, notes Crombie.
“We have found that our grantees have struggled to move their innovation out of the lab and into clinical trials because material for clinical trials needs to be manufactured to a high standard,” she explains.
“There are a limited number of sites in the world capable of making this material and because academics doing a Phase I or Phase II trial are often not looking for large amounts of product, the commercial sites will routinely prioritize the more lucrative larger commercial scale batch manufacturers.
“Consequently, academic researchers developing gene therapies, often funded by grants from charities or research councils, struggle to get the material they need. When they do, it is expensive and they often find themselves bumped to the back of the back of the queue if a bigger, more lucrative contract comes along.”
Academic gene therapy developers often lack the technical know-how needed to manufacture supplies for trials, so they tend to rely on contractors. This reliance further stretches limited capacity and slows the development of expertise, Crombie says.
“This gap in the translational pathway results in delay, increased costs, and a loss of knowledge and skills needed to grow and sustain this sector and is hampering the entry of new gene therapies into clinical application,” she continues.
“The new Innovation Hubs will give academics across the U.K. access to the skills, materials, and manufacturing capacity they need, within a reliable timescale and cost which will accelerate the access of patients to therapeutic solutions to rare and life-threatening diseases.”
Limited vector capacity is often cited as a major challenge for gene therapy firms. In the past few years various researchers, industry groups, and observers in the mainstream press have suggested a lack of vectors was slowing development of the sector.
The new network will aim to address this, according to Crombie, who points out, “The main focus of the Hubs will be viral vector manufacture. The Hubs will be looking to establish reliable manufacture and will also look at new technologies that can improve their processes.”
Often the reliance on complex adherent culture technologies for vector production is cited as a hurdle. However, while this is a factor, the real problem is the high level of demand, according to Crombie.
“There is a global shortage in capacity, for the production of vectors and it is a challenge for gene therapy developers. Production of gene therapies is highly skilled and time-consuming process—while some technologies use adherent cells, it’s not the main issue,” she says.
“We have been witnessing an exponential rise in gene therapy products reaching clinical trials in recent years. Many of the products are driven by academics or academic spin-out companies, who do not have in house manufacturing capability. The number of manufacturing plants or CROs has simply not kept pace with the number of gene therapies that need manufacturing.”