Phacilitate Leaders Forum

17th - 18th SEPTEMBER 2019

ROYAL LANCASTER LONDON

European Success Stories

European Success Stories

EUROPEAN SUCCESS STORIES

We are delighted to share with you some of our favourite European advanced therapy Success Stories. One of the aims of Phacilitate Leaders Europe is to advocate and promote Europe as a region of opportunity for this industry, as we strive to improve the standard of healthcare for the patients that need these therapies.
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Our Success Stories are intended to inspire and encourage faith in Europe. Enjoy!

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Zynteglo

  • WHO: bluebird bio
  • WHEN: June 2019
  • WHERE: Amsterdam
  • STATUS: Approved by the EMA
     

The buebird therapy for beta-thalassaemia (a rare inherited blood condition that causes severe anaemia) addresses an unmet medical need.

As such it benefited from support within the PRIME scheme, EMA’s platform for early and enhanced dialogue with developers of promising new medicines. This interaction led to a more robust application package which allowed accelerated assessment of Zynteglo in 150 days, the fastest review time for an ATMP to date.

Find out more about EMA PRIME here

Find out more about Zynteglo here


 

Collaboration between TCR2 Therapeutics and Cell & Gene Therapy Catapult

  • WHO: TCR2 Therapeutics and Cell & Gene Therapy Catapult
  • WHEN: April 2018
  • WHERE: Stevenage, UK


TCR2 Therapeutics, is to become the first US organisation to work with the Cell and Gene Therapy (CGT) Catapult at its Stevenage manufacturing centre.

TCR2 has selected the facility to establish a site for global manufacturing and capability development to advance novel T cell receptor therapies for patients suffering from cancer.

By choosing to work with the Cell & Gene Therapy Catapult manufacturing centre, TCR2 Therapeutics demonstrates to the world that the UK is a global leader in this sector.

Find out more about Cell & Gene Therapy Catapult

Find out more about TCR2 Therapeutics

Find out more about the collaboration here


 

Gamida-Lonza

WHO: Gamida Cell & Lonza
WHERE: Israel & the Netherlands
WHEN: June 2019
STATUS: Phase 111 trials

Gamida Cell (an Israel-based biopharma company) and Lonza have had the nod of approval to continue with their manufacturing agreement for Gamida's gene therapy, Omidubicel. The Israel-based Gamida partnered with Lonza for the future commercial production of the therapy, which is currently in Phase 111 trials. 

Lonza will be building infrastructure for the production of Omidubicel at their facility in Geleen, in the Netherlands, which may be expanded in the future should demand for the therapy increase. 

Find out more about Gamida Cell 

Find out more about Lonza Pharma & Biotech 

Find out more about Omidubicel


 

CRISPR Therapeutics & Vertex Pharmaceuticals

WHO: Vertex Pharmaceuticals & CRISPR Therapeutics
WHERE: Switzerland & USA
WHEN: 2015 - Present
STATUS: Phase 1/2 trials

The current collaboration between Vertex Pharmaceuticals (US) and the Swiss company CRISPR Therapeutics is to be expanded in 2019. The €890M deal will enable the two companies to continue to develop their treatment for a variety of genetic diseases, one of which causes muscle weakness, using the gene editing tool CRISPR/Cas9. 

The collaboration began back in 2015, when the partners joined with the intention of using CRISPR/Cas9 to treat other genetic diseases such as sickle cell disease and cystic fibrosis. Trials for these therapies are currently in Phase 1/2. 

Find out more about CRISPR/Cas9 and the partnership


 

Nightstar Therapeutics Acquisition

WHO: Biogen & Nightstar Therapeutics
WHERE: United Kingdom
WHEN: March 2019

In March this year, Syncona's Nightstar Therapeutics agreed to be acquired by US Biotech giant Biogen. The deal, worth $800M, gives Biogen a clinical pipeline of gene therapies in opthalmology.

Nightstar focuses primarily on adeno-associated virus (AAV) treatments such as NSR-REP1 which treats genetically inherited retinal disorders. If un-treated, these disorders can result in blindness, and these therapies are designed as one-time only treatments.

Find out more about Nightstar Therapeutics


 

$5.1bn Gilead & Galapagos Deal

WHO: Gilead Pharma & Galapagos
WHERE: Belgium
WHEN: July 2019
STATUS: Phase 3 trials 

A groundbreaking deal was just announced between Gilead Sciences, Inc. and Galapagos NV, a Belgian biotech, to develop rheumatoid arthritis and inflammatory disease drugs. The deal, worth $5.1bn, will see the US pharma giant pay $3.95bn upfront, and the remaining $1.1bn in equity investment. 

The deal will give Gilead access to Galapagos' phase 3 candidate for IPF (idiopathic pulmonary fibrosis), GLPG1690.

Both parties will co-fund and share the costs for all projects going forward. 

Find out more about Gilead

Find out more about Galapagos

View the full press release and find out about the previous Gilead/Galapagos deal


 

Would you like to share your European Success Story?

Have you had a recently approved therapeutic? Would you like to shout about your recent partnership or collaboration?

Get in touch with us about your news and you could be featured here too!

Testimonials
  • Real thinking forward combining experts in the field from manufacturing, regulatory and value vision” – “great network with strong experience and sharing risks & opportunities
    Jean-Philippe Combal
    GenSight Biologics
  • The conference consistently proves to be a great forum for networking with industry leaders in the development of cellular and gene therapies as well as vendors for important technologies and services.
    Michael Covington
    Juno Therapeutics, Inc
  • It was a very interesting event as all participants, including speakers were ready to exchange, even on the most challenging subjects such as reimbursement, GMP guidance
    Bertrand Alexandre
    Celyad
  • Phacilitate EU delivered it's promise and more, the topics discussed were highly relevant, with very good and professional speakers and panelists. You will see me next year in Berlin.
    Dr Ohad Karnieli
    Karnieli Ltd