Genetic Technology
(TechVision)
D758-TV
January 8, 2016
Innovations across Gene Therapy,
Infectious Diseases, and
Neurodegenerative Diseases
Contents
Section Slide Numbers
Research Collaborations for Gene Therapy 3
AGTC and Synpromics Collaborate for Developing Gene Therapies for Eye Diseases 4
Annapurna Therapeutics and Weil Cornell Collaborate for Developing Gene Therapies 5
Innovations in Infectious and Neurodegenerative diseases 6
Scientists Identify a Role for Host Ligase in the Pathogenesis of Influenza A 7
Biopharmaceutical Company Actively Involves in Solving Growing Threat
of Alzheimer’s Disease 8
Appendix 9
Patent Analysis 10
Industry Interactions 12
3
Research Collaborations for Gene Therapy
4
AGTC and Synpromics Collaborate for Developing Gene Therapies for
Eye Diseases
Unmet Needs
Innovation Profile
Potential
Applications
Future Perspectives …
The advancement of gene therapy applications is currently limited
by the availability of strong promoter elements that facilitate
optimal expression of the target gene.
Natural biological promoters are not ideally suited for clinical
applications in the biotechnological sector.
…
Funding • …
Collaborative Venture
What
Where Who
A broad, multi-target joint venture for
developing adeno-associated virus (AAV)-…
AGTC, is an American
biotech firm based in
Florida, United States. …
Applied Genetic Technologies
Corporation (AGTC) is a
biotech firm that develops gene
therapies for eye diseases.
….
Novel technologies are required to optimize gene therapies that
are clinically effective at lower doses.
• ….
5
Innovations in Infectious and Neurodegenerative
Diseases
6
Scientists Identify a Role for Host Ligase in the Pathogenesis of
Influenza A
Funding The study was supported by:
• …
Potential Applications • …
Future Perspectives • …
Overview Analytical Findings
Innovators Technology
Advancements in “OMICS’
analyses have created a
large repository of datasets
for the study of host-pathogen
interactions during influenza
A infection. …
….
The research team has created a user-friendly Web portal of the integrated
data that elucidates the biochemical landscape of viral-host interactions:
www.metascape.org/IAV
• ….
…..
7
Appendix
8
Patent Analysis Novel patents for treating rare diseases
Title Publication
Date/Publication
Number
Assignee Inventor Abstract
Source: Frost & Sullivan
Rare diseases is a disease segment with multiple market opportunities. Currently, there are a plethora of different therapeutic techniques
being developed specifically to tackle rare diseases, including, gene therapy for rare genetic disorders, cancer immunotherapy for rare
cancers, RNAi therapy, as well as small molecular and monoclonal antibody-based therapeutics. In 2013, a company working in the
gene therapy space garnered approval for its Glybera therapy, targeted toward lysosomal storage diseases. Similarly, companies such
as Spark Therapeutics have been working toward the development of therapies for rare ophthalmic disorders. However, since the market
is very small and niche, the drugs that are developed seem to be at extremely high price points. This results in only one company as the
market leader for treating a specific rare disorder. Hence, it is vital that the current patent approvals are garnered for the methodologies
that have been developed to treat these diseases, so that companies are able to leverage their technologies in the pursuit of developing
these therapeutics.
9
Industry Interactions