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Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium, Imperial Innovations and Oxford Biomedica Announce New Partnership to Develop First-in-Class Gene Therapy for Cystic Fibrosis

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  • This new collaboration unites leaders in cystic fibrosis gene
    therapy, lentiviral development and production, and the treatment of
    respiratory diseases to develop the first gene therapy for cystic
    fibrosis.
  • Boehringer Ingelheim steps into the field of gene therapy, a focus
    area of its Research Beyond Borders initiative, with experienced
    partners, in an effort to bring to cystic fibrosis patients a
    long-term treatment aimed at correcting the underlying genetic causes
    of this devastating disease.
  • Boehringer Ingelheim will collaborate with the UK Cystic Fibrosis
    Gene Therapy Consortium on research and development and has option and
    license agreements with Imperial Innovations and Oxford BioMedica on
    the viral vector as well as production technology, while Oxford
    BioMedica will manufacture the viral vector; the UK CF Gene Therapy
    Consortium will lead the development and completion of the preclinical
    studies and bring together a package to support first-in-man clinical
    trials.

Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium
(GTC)—consisting of Imperial College London and the Universities of
Oxford and Edinburgh—Imperial Innovations, and Oxford BioMedica (OXB)
announce a global collaboration to develop a first-in-class, long-term
therapy for patients with cystic fibrosis (CF). The new partnership
brings together the academic partners' leading expertise in developing
gene therapy for CF and OXB's leading expertise in manufacturing
lentiviral vector-based therapies with Boehringer Ingelheim's
capabilities in drug discovery and the clinical development of novel
breakthrough therapeutic agents.

Cystic fibrosis is a genetic disease that causes persistent lung
infections and usually limits a person's ability to breathe over time.
There are more than 2,000 known mutations in the gene for the cystic
fibrosis transmembrane conductance regulator (CFTR), many of which
result in a person developing CF. The disease is estimated to affect
70,000 people worldwide, with the majority of those located in the US
and Europe. Existing treatments and those under development can only
slow the progression of disease. As such, CF continues to be associated
with considerable morbidity, mortality, and a high burden of therapy. In
addition, existing treatments will have varying effects depending on a
patient's mutation status, as determined by the particular gene
variation that causes their disease. Thus, there is a critical unmet
need for therapies that address all CFTR mutations; gene therapy has the
potential to provide such a solution.

The collaboration will focus on a novel approach using a
replication-deficient lentiviral vector in an inhaled formulation, to
introduce a healthy copy of the CFTR gene into the cells of the lung.
This method has demonstrated high gene transfer efficiency and offers
the possibility of repeated administration to maintain the therapeutic
effect. Gene therapy is the only therapeutic approach to date that can
address all CFTR gene mutations, thus potentially offering a universal
treatment option.

Professor Eric Alton, Coordinator of the UK Cystic Fibrosis Gene Therapy
Consortium, said "The UK CF Gene Therapy Consortium has, for the last 17
years, vigorously sought to establish whether gene therapy can become a
clinically viable option for patients with CF. From the beginning the
GTC identified that this goal would require incremental increases in
knowledge. We have, therefore, built on our non-viral gene therapy
experience to develop a new viral vector-based product, which is
currently funded by the Health Innovation Challenge Fund (a partnership
between the Wellcome Trust and the Department of Health and Social Care)
and the Cystic Fibrosis Trust. It is with great pleasure that we now
join forces with two world-class organisations. Boehringer Ingelheim
will provide its multinational industry expertise, including a rich
heritage in the respiratory field, to drive the product towards the
clinic, whilst Oxford BioMedica is the acknowledged leader in the field
of lentiviral vector manufacturing. The GTC believes that this
partnership provides CF patients with the optimal chance to establish
gene therapy as routine clinical practice, relevant to all patients,
irrespective of their mutation status, and in due course to both prevent
lung disease as well as treat established problems. We would like to
take this opportunity to warmly thank all of our fundraisers who have
supported us over many years."

"This novel three-way partnership brings together an unparalleled
combination of clinical, scientific, manufacturing and commercial skills
in an effort to develop new treatments and make a major contribution to
the lives of patients affected by cystic fibrosis," stated John Dawson,
Chief Executive Officer of Oxford BioMedica. "The GTC has been working
determinedly for over 15 years to get to this exciting point of forming
a partnership with Boehringer Ingelheim, a global pharmaceutical company
with respiratory expertise. Our contribution to this partnership
reaffirms our leading position in the development and manufacturing of
lentiviral vector gene therapy products at large scale. We look forward
to working with our new academic and industry partners."

"Through this collaboration, we are joining forces with some of the top
talents in this disease space to propel treatment advances forward,"
said Clive R. Wood, Ph.D., Senior Corporate Vice President, Discovery
Research at Boehringer Ingelheim. "Bringing together our existing
expertise as a leader for nearly a century in the discovery and
development of therapies that have advanced patient care in respiratory
diseases with the gene therapy knowledge of our partners, we aim to
unlock unprecedented opportunities for patients with this devastating
disease, who are desperately waiting for better treatment options."

Boehringer Ingelheim has received an option to license the exclusive
global rights to develop, manufacture, register, and commercialise this
lentiviral vector-based gene therapy for the treatment of cystic
fibrosis. Financial terms are not disclosed. During the option period
the partners will work closely together to pursue the development of
this innovative approach financed by Boehringer Ingelheim. The new
collaboration is a joint initiative of Boehringer Ingelheim's
Respiratory Therapeutic Area and Research Beyond Borders (RBB), which is
one of the pillars of Boehringer Ingelheim's research and development
strategy and explores emerging science, disease areas and technology. It
builds on Boehringer Ingelheim's commitment to early science and its
comprehensive approach to respiratory research and development to
investigate new treatments that have significant clinical value in areas
of high unmet need.

Andrew Tingey, Director of Healthcare Licensing at Imperial Innovations
said, "The UK Cystic Fibrosis Gene Therapy Consortium shows the power of
world-class academic groups collaborating to develop advanced potential
therapies. We were delighted to have been chosen as the technology
transfer partner for the GTC and have worked closely with them during
the development of this potential new gene therapy, securing the
intellectual property necessary to drive forward its commercial
development and to support the collaboration with Boehringer Ingelheim
and Oxford BioMedica. The combination of expertise and resources
realised by this deal will give the project a unique opportunity to
develop an advanced therapy that could significantly impact the lives of
thousands of people living with cystic fibrosis, and we are delighted to
have played the lead role in securing this partnership."

Please click on the following link for ‘Notes to Editors':

http://www.boehringer-ingelheim.com/press-release/new-partnership-develop-gene-therapy-cystic-fibrosis

The information contained within this announcement is deemed by the
Company to constitute inside information as stipulated under the Market
Abuse Regulation (EU) No. 596/2014. Upon the publication of this
announcement via the Regulatory Information Service, this inside
information is now considered to be in the public domain.

Media Contacts:

Boehringer Ingelheim
Dr. Reinhard Malin
Head of
Communications, Innovation Unit
Boehringer Ingelheim Corporate
Center GmbH
Media + PR
P: +49 6132 77-90815
reinhard.malin@boehringer-ingelheim.com

Linda Ruckel
Associate Director, Media and Corporate
Reputation
Boehringer Ingelheim US
Media + PR
P: +1
203-791-6672
linda.ruckel@boehringer-ingelheim.com

UK Cystic Fibrosis Gene Therapy Consortium
Imperial
College London

Professor Eric Alton
P:
+44-207-594-7937
e.alton@ic.ac.uk

Professor Uta Griesenbach
P: +44-207-594-7927
u.griesenbach@ic.ac.uk

Professor Jane Davies
P: +44-207-594-7973
j.c.davies@ic.ac.uk

Tracy Harman
P: +44-207-594-7932
t.harman@ic.ac.uk

University of Oxford
Professor
Deborah Gill

P: +44-1865 221845
deborah.gill@ndcls.ox.ac.uk

Associate Professor Stephen Hyde
P: +44-1865 221845
steve.hyde@ndcls.ox.ac.uk

University of Edinburgh
Dr
Christopher Boyd

P: +44-131-651-8733
Chris.boyd@ed.ac.uk

Dr Gerry McLachlan
P: +44-131-651-9162
gerry.mclachlan@roslin.ed.ac.uk

Imperial Innovations
+44 (0)20 2053 8850
Gavin Reed
Marketing
Manager
Gavin.reed@imperialinnovations.co.uk

David Barreto Ian
Marketing Executive
dbi@imperialinnovations.co.uk

Oxford BioMedica

Oxford BioMedica plc:
John Dawson,
Chief Executive Officer
Tel: +44 (0)1865 783 000
Stuart
Paynter, Chief Financial Officer

Financial and corporate communications enquiries:
Consilium
Strategic Communications
Tel: +44 (0)20 3709 5700
Mary-Jane
Elliott/Matthew Neal/Olivia Manser/Laura Thornton

Peel Hunt (Joint Corporate Brokers):
Tel:
+44 (0)20 7418 8900
James Steel
Christopher Golden

WG Partners (Joint Corporate Brokers):
Tel:
+44 (0)20 3705 9321
David Wilson
Claes Spång

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