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