by Natalie Morrison
LONDON, May 5 (APM) - GlaxoSmithKline is investing "significantly" in the cell and gene therapy space, seeing it as a way to treat common diseases in the future, Emily Culme-Seymour, external strategy manager of Gene Therapy at GlaxoSmithKline's Rare Diseases Unit has told APM.
The company is currently working on cell and gene therapies for rare diseases. Speaking on the sidelines of International Society for Cellular Therapy's (ISCT) annual meeting in London on Thursday, Culme-Seymour said the rare disease space is a "fantastic starting point" to learn how to deliver these therapies.
However, she said in the long term, GSK will look to expand its cell and gene therapy platform to treat other, more common diseases.
"We are building a big platform for cell and gene therapies and investing significantly because we see this as a way to deliver to more common diseases in the future," she said.
She stressed the rare disease space still holds "huge unmet medical need", noting GSK remains "fully committed" to it and views it as a key business area.
Culme-Seymour's comments are the latest confirming GSK's interest in the field and belief in its growth potential. In September 2016, following Strimvelis' approval, both Culme-Seymour and head of gene therapy development Sven Kili told a Bio Industry Association (BIA) webinar that cell and gene therapies are becoming a "viable grown-up" alternative to small molecules and antibodies (APMMA 49462).
GSK has been widely recognised as one of the few big pharma making a commitment to cell and gene therapy. That is with the exception of a few others including Novartis, which is lined up to be one of the first to have a CAR-T cell therapy - CTL019 (tisagenlecleucel-T) - approved in the U.S. this year.
Asked why big pharma are seemingly holding back, Culme-Seymour said it could come down to risk aversity and a desire to see a demonstration of commercialisation from another player before moving.
"Sometimes it's easiest to be the second through," she said.
Other firms could still be considering where best to place their investment in terms of vector types and whether they go for an autologous or allogeneic therapy - made from the patients' own tissue or made from donor tissue - she said.
GSK has used lentivirus vector and autologous.
At the conference, a long-standing debate continued over whether safety and efficacy benefits of autologous therapy outweigh the convenience and scalability of allogeneic therapy when it comes to commercialisation.
GSK had its first gene therapy product Strimvelis approved in Europe in May last year for the rare paediatric immune deficiency disease ADA Severe Combined Immune Deficiency (ADA-SCID) (APMMA 48052). The product is autologous.
Asked why GSK chose autologous production, Culme-Seymour said that "autologous has shown the efficacy". She added that the autologous gene therapy space in general has seen "exciting results in clinic" and that the decision was based on what was most promising for patients.
In terms of commercialisation, she said that scalability and having a bank of allogeneic cells available for production is obviously easier, but insisted if the "daunting list" of autologous challenges are overcome there is no reason an autologous product cannot be viable.
The company has since been working on its long-term strategy for selling Strimvelis, which is reportedly priced at 594,000 euros for a single infusion. Kili has previously said its pricing strategy may mean it "may not make a profit" (APMMA 50134).
The disease life-threatening immune deficiency affects approximately 15 children a year in Europe, according to GSK.
GSK has primarily worked in cell and gene therapy through collaborations, Culme-Seymour added, citing its 2010 Tiget partnership deal including Italian biomedical charity Fondazione Telethon and St Raffaele hospital in Milan.
Under the R&D deal, the firm got rights to develop and commercialise seven gene therapies, Culme-Seymour added. Strimvelis was the lead programme from the tie-up.
Other programmes from the collaboration including for rare diseases Metachromatic Leukodystrophy (MLD) and Wiskott Aldrich Syndrome, are both autologous gene therapies in late-stage development, Culme-Seymour said.
GSK also signed a partnership deal with Adaptimmune, under which it is investigating engineered T Cells, she said. The deal includes development of Adaptimmune's lead clinical cancer programme, the affinity enhanced T Cell immunotherapy GSK3377794 (APMMA 46174).
/nm/nh
TRY APM Market Access AND GET ACCESS TO THE FULL CONTENT
Interviews with KOLs/senior executives amongst the Regulators, Payers, Health, Medical & Pharmaceutical organisations
Events coverage with a unique focus on Market Access & sustainability of healthcare systems
6 European bureaus : Berlin, Brussels, London, Madrid, Milan & Paris
Ask for a Free trial and get access to the latest stories
Our coverage includes:
- Health Care
- Market Access
- HTA – policies & practices
- European medicine regulations
- Drug safety issues
- Pricing & Reimbursement
- International medicines agencies
If you are a Payer, Pharmaceutical or Consulting professional our premium data will keep you informed on the regulatory, pricing, market access and cost-effectiveness issues that impact all stakeholders.
REQUEST
an initial 10 day temporary access of APM Market Access.
