Targeted Genetics Corporation is committed to the development and commercialization of innovative therapies for the prevention and treatment of diseases with significant unmet medical need. Our efforts focus on utilizing an Adeno-Associated Virus (AAV) technology platform to deliver therapeutic constructs that increase or decrease gene function. We hold AAV-based assets for therapeutic program efforts and control important intellectual property rights around AAV serotypes, certain therapeutic uses of AAV and intellectual property and know-how relevant to AAV manufacturing. In addition to certain early stage pre-clinical program development efforts, we are currently developing, through collaborations, product candidates in Leber’s congenital amaurosis (LCA), a form of blindness, heart failure and Huntington’s disease (HD), a neurological disease.
LCA:
Our leading product development efforts focus on the investigation of a clinical-stage AAV-based product candidate for treatment of Leber’s Congenital Amaurosis (LCA) – an ocular disease that leads to blindness due to the effects of a mutation of the RPE65 gene. Utilizing AAV vectors to deliver the RPE65 gene, this product candidate is currently under evaluation in subjects in a Phase 2 dose escalation clinical trial. It is anticipated that interim data will be available from this trial at several different points of time throughout 2010, and that final data will be complete by the end of 2010. This product candidate is being developed in collaboration with Robin Ali, Ph.D., at the University College London/Moorfields Eye Hospital.
Heart Failure:
Under license and manufacturing agreements, Celladon Corporation utilizes proprietary AAV vector technology from Targeted Genetics for the development of MYDICAR® – a genetically-targeted enzyme replacement therapy for advanced heart failure. MYDICAR is currently under evaluation in subjects in a Phase 1/2 clinical trial. We manufactured Celladon’s MYDICAR product candidate for phase 1, 2 and future phase 3 clinical studies. We have transferred technology to enable Celladon to manufacture MYDICAR in the future through contract manufacturing organizations or a commercial partner, and if the program progresses further through clinical studies and through the FDA approval process, we could receive significant milestone payments and royalties.
HD:
Preclinical studies focused on identifying a candidate to develop a therapeutic for the treatment of Huntington’s disease (HD) continue in the laboratory of Beverly Davidson, Ph.D., at the University of Iowa (UI). HD is an incurable neurodegenerative disorder that results from mutations to the gene that codes for the huntingtin protein. Based on preclinical data collected over the last year, scientists at UI are performing additional research efforts with the goal of identifying a product candidate to put on a development path. We have a licensing relationship with UI around certain intellectual property resulting from Dr. Davidson’s research efforts.
Inflammatory Arthritis:
In 2008 we completed a Phase 2 clinical trial of tgAAC94 for the treatment of inflammatory arthritis, which included 127 patients in a randomized placebo controlled trial. The trial data demonstrated that tgAAC94 was well tolerated and showed preliminary signs of efficacy, most notably a 30% decrease in patient-reported pain and functional scores. Based on our currently available resources, we will initiate additional clinical studies of tgAAC94 only with additional external funding from a development and commercialization partner.
HIV/AIDS Vaccines:
Preclinical development for the HIV/AIDS vaccine development project, which was funded by the National Institute of Allergy and Infectious Diseases, and under subcontract with Children’s Hospital of Philadelphia and Nationwide Children’s Hospital, has been completed. Our preclinical development and manufacturing of material for clinical trials under this contract is now complete as the program is entering into clinical trials which will be funded and managed by NIAID.
ALS:
We have discontinued preclinical development of a small-molecule-based product candidate to treat amyotrophic lateral sclerosis (ALS), which was under development with collaborator John Engelhardt, Ph.D., at the University of Iowa.
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