Cambridge, MA – March 12, 2010 – Genetix Pharmaceuticals, a leading gene therapy company
developing breakthrough treatments for severe genetic disorders, announced today that it
has completed a $35 million Series B financing with new investors Third Rock Ventures and
Genzyme Ventures joining TVM Capital, Forbion and Easton Capital. Proceeds from the financing
will be used to advance current clinical programs, strengthen platform capabilities and further
expand the team.
“The Genetix platform represents the convergence of the powerful therapeutic modality of gene therapy
with a class of severe genetic disorders lacking safe or widely available treatment options,” said Nick Leschly,
Interim President of Genetix and Partner of Third Rock Ventures. “Genetix not only has a compelling value
proposition, but also holds great potential to make a difference for patients. We are very encouraged by the
promising clinical data in both Adrenoleukodystrophy (ALD) and beta-Thalassemia showing arrested disease
progression in a small number of patients treated to date, and by the opportunity to build a best-in-class
company in the field of gene therapy for severe diseases.”
“Genetix is making important progress in its gene therapy clinical development programs,” said Alan Walts,
Managing Director of Genzyme Ventures. “We look forward to joining the company's Board, supporting its growth,
and bringing to bear our experience in rare genetic diseases.”
Gene Therapy Resurgence
Over the last few years, a small but growing number of gene therapy trials have yielded increasingly promising results.
Genetix’s proprietary lentiviral technology delivers corrective genes to the patient’s own bone marrow cells,
providing a one-time effective therapy, a true paradigm shift in the treatment of genetic diseases. Genetix
has programs in clinical development in Adrenoleukodystrophy (ALD), a severe neurodegenerative disorder,
and beta-Thalassemia, one of the most prevalent human genetic disorders. Both of these programs have shown
strong early results to date and stabilization of their respective diseases. Results from the ALD program
have been published in a November 2009 issue of Science. To view the article, please click on the following link:
http://www.sciencemag.org/cgi/content/abstract/326/5954/818
“Having been in the gene therapy field for over 25 years, it is great to see the new groundswell of excitement from
the scientific community and the pharmaceutical industry, reflecting both technology advances and early, but exciting,
clinical data,” said Mitchell Finer, Ph.D., Chief Scientific Officer of Genetix Pharmaceuticals. “This financing puts
us in a great position to expand our team, accelerate the development of our later stage products and further build our
unique gene therapy platform so we can pursue our vision of delivering novel, breakthrough treatments for patients.”
Experienced Team
Genetix Pharmaceuticals is led by an experienced team with a proven track record of building
successful life sciences companies. The new members of the management team include Nick Leschly
from Third Rock Ventures as Interim President; Mitchell Finer, Ph.D. as Chief Scientific Officer;
Phil Reilly, M.D. from Third Rock Ventures as Interim Chief Medical Officer; and Anup Arora from
Easton Capital, who is continuing to lead the business development and operations activities on
an interim basis. In parallel with the financing, the current Chief Executive Officer, Alfred
Slanetz, Ph.D. will depart from the company in order to pursue other opportunities in the biotech
field. Dr. Slanetz has been a key contributor at Genetix and a driver of the Company’s
accomplishments over the past few years. In addition, the current VP of Corporate Development
and Co-Founder, Ron Dorazio, M.D. will transition into a strategic advisor role for the company.
Dr. Dorazio’s contributions have been fundamental since the inception of the company both scientifically
and strategically.
The new Board of Directors will consist of Jim Geraghty of Genzyme; Axel Polack, M.D. of
TVM Capital; Geert-Jan Mulder, M.D. of Forbion and Nick Leschly of Third Rock Ventures.
The Scientific Advisory Board will continue to be led by Genetix founder Dr. Philippe
Leboulch, along with Dr. Patrick Aubourg.
About Genetix Pharmaceuticals
Genetix Pharmaceuticals is a leading gene therapy company developing breakthrough treatments
for severe genetic disorders. Its products treat the underlying cause of genetically based diseases
by delivering corrective genes to the patient’s own bone marrow, providing the potential for a one-time
transformative treatment. Genetix's lead clinical product in development is Lenti-D™ for
Adrenoleukodystrophy (ALD), also known as Lorenzo's Oil disease. The results of the ongoing ALD trial,
based upon work conducted by the National Institute of Health and Medical Research (INSERM), were named
to Science magazine’s "Scientific Breakthroughs of 2009." In addition to ALD, Genetix is conducting
Phase I/II trials with its LentiGlobin™ product in beta-Thalassemia. To date, all patients receiving
the full therapy with both products have shown disease arrest. In addition to Genetix’s lead programs,
the company’s proprietary stem cell processing, GMP manufacturing and gene therapy vector technologies
are applicable to treating other genetic diseases, opening the door to the development of additional therapeutics.
Genetix is privately financed by an experienced syndicate of healthcare investors, including Third Rock Ventures,
TVM Capital, Forbion Capital Partners, Easton Capital and Genzyme Ventures. Its operations are located in
Cambridge, MA and Paris, France. For more information, please visit http://www.genetixpharm.com/.
Contact:
Lisa Rivero
Director of Media Relations
LaVoie Group
Tel: 978-745-4200 X 106
lrivero@lavoiegroup.com
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