9:10         Alternative Delivery of Nanobodies

Josi Holz, Ph.D., Chief Medical Officer, Ablynx nv

NanobodiesÒ are the smallest functional fragment of heavy chain only   Llama derived   monoclonal antibodies. Protein engineering and formatting transforms these NanobodiesÒ in powerful tools in the diagnostic and treatment of various diseases. The inherent stability of the molecules makes them ideal candidates for exploratory studies on alternative ways of drug delivery through the skin (SC), orally and pulmonary to name a few. The data presented will review the capabilities of NanobodiesÒ administered beyond the intravenous injection route.

9:40         Site-Specific PEGylation to Improve the PK/PD Properties of Biologics

Jason Pinkstaff, Ph.D., Associate Director, Preclinical Science, Ambrx, Inc.

Ambrx uses an expanded set of amino acids to address the limitations intrinsic to the 20 natural amino acids. Our approach, termed protein medicinal chemistry™, combines the power of medicinal chemistry with recombinant biosynthesis. Through the application of Ambrx’s proprietary technology, numerous variants of the naturally occurring wild-type protein can be generated, with each variant containing a single Ambrx Amino acid incorporated into the protein backbone. A single polyethylene glycol (PEG) molecule can then be attached site-specifically to the incorporated Ambrx amino acid. Molecules that vary only in the site of PEG attachment can exhibit significant differences in pharmacology, receptor binding, efficacy and biophysical stability.

10:10       Coffee Break in the Exhibit Hall

11:10       Systemically Acting Chemotherapeutic Enzyme Engineered for High Activity, Serum Stability and in Vivo Half-Life

George Georgiou, Ph.D., Department of Chemistry and Biochemistry, University of Texas, Austin

This presentation will describe the development and preclinical evaluation of engineered human therapeutic enzymes for amino acid depletion therapy in cancer.  Protein engineering approaches were employed to engineer enzymes with high catalytic activity and low immunogenicity.  Additionally, we have devised new approaches for increasing the half life of these proteins and for achieving optimal pharmacodynamic profiles for  cancer treatment.

11:40       Oral Delivery, Strongly Reduced Degradation and Strongly Enhanced Efficacy Resulting from Non-Native Thioether-Bridged Amino Acids, Enzymatically Introduced in Therapeutic Peptides

Gert Moll, Ph.D., Project leader, Biomade Technology Foundation

We stabilized therapeutic peptides by introducing thioether bridges. The resulting peptides can be delivered orally, pulmonarily and subcutaneously,  are proteolytically resistant, have up to 100-fold higher in vivo concentration in blood plasma of rats, have strongly enhanced receptor interaction and strongly enhanced therapeutic potential.

12:10       Luncheon Presentation I (Opportunity Available)

12:40       Luncheon Presentation II (Opportunity Available)

1:10         Break

LOCAL DELIVERY OF AGONISTS

1:30         Chairperson’s Remarks

Tudor Arvinte, Ph.D., Chairman, Chief Executive Officer, Therapeomic Inc. and Department of Pharmaceutics and Biopharmaceutics, School of Pharmacy Geneva-Lausanne, University of Geneva

1:35         Relevance of Transforming Growth Factor Beta 3 (TGF-ß3) for Local Therapies

Tudor Arvinte, Ph.D., Chairman, Chief Executive Officer, Therapeomic Inc. and Department of Pharmaceutics and Biopharmaceutics, School of Pharmacy Geneva-Lausanne, University of Geneva

One of the most promising ways to stimulate tissue repair is the application at the injured site of growth factors. TGF-ß3 is an important mediator of growth, maintenance and repair processes in human cells. TGF-ß3 is abundant in bone matrix and was shown to stimulate bone and connective tissue formation as well as the growth of blood vessels. Beside the right choice of the growth factor, the success of local  repair therapies is very much dependent on the formulation of the growth hormone formulation and the delivery system. Optimal formulations of TGF-ß3 were developed based on the understanding of the molecules’ biophysical and biochemical properties and by in vivo optimization using relevant animal models.  Data will be presented on the use of TGF-ß3 in local bone repair and in the local stimulation of angiogenesis and vasculogenesis.

2:05         Local Delivery of Drugs Into the Eye Using Electroporation

Elodie Touchard, Centre de Recherche des Cordeliers, René Descartes University, Pierre et Marie Curie University

We have developed an innovative local protein delivery strategy for the eye. The ciliary muscle is a smooth muscle responsible for accommodation. This strategy is based on the use of ciliary muscle as the target of non viral gene transfection, resulting in an intraocular bioreactor that is able to secrete any therapeutic protein in the ocular media on the long term.  Preclinical studies in rat models of ocular diseases will highlight the therapeutic potential of this new concept which may become a useful alternative to conventional current therapies, especially for the management of the most common ocular diseases leading to blindness in patients.

2:35         Sponsored Presentation
Title and speaker to be announced   Sponsored by

3:05         Refreshment Break in the Exhibit Hall

3:50         Novel Polymers for Drug Delivery

Brian Carlin, FMC Corporation 

4:20         To be Announced

4:50         Networking Cocktail Reception in the Exhibit Hall

6:00         Close of Day

For questions, please contact:
Christina Lingham
Executive Director, Conference
Cambridge Healthtech Institute
Fax: 781-972-5425
E-mail: clingham@healthtech.com 

For sponsorship and exhibit information, contact:
Carol Dinerstein
Cambridge Healthtech Institute
Phone: 781-972-5471
E-mail: dinerstein@healthtech.com