ペプチド及びタンパク質由来の治療法に関する会議 - 3日目

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アジェンダ (英語PDF)

Wednesday, January 9

7:00am - 5:30 pm Registration Open

7:30 - 8:15 Breakfast Workshop 

8:15 Chairperson’s Remarks

8:20 New Insights into Biolipids-Protein Interactions: The Case Study of the Binding Features of Lysophospholipids 
Genevieve Hansen, Ph.D., Vice President of Research, Lpath Therapeutics
Lpath Therapeutics has developed a unique technology platform to generate monoclonal antibodies (mAb) against lipid-based targets. The example of the development of a therapeutic mAb to the biolipid Sphingosine-1-Phosphate (S1P) involved in angiogenesis and tumor growth will be presented. Computational modeling and mutagenesis studies have revealed the key residues involved in the binding interaction between S1P and the mAb. Interestingly, the binding pockets have similar structural features than the ones of the G protein-coupled receptors interacting with S1P and other biolipids. These findings are of general relevance as they lay the foundation for the development of therapeutics targeting biolipids.

8:50 Second Generation Affibody Molecules
Maria Akerman, Ph.D., Project Manager, Affibody AB 
Affibody molecules are small binding proteins that have been successfully used for tumor targeting both for molecular imaging and therapy. Clinical data in molecular imaging shows the same rapid clearance as in animal models. Both binding and other properties have been engineered to obtain molecules suitable for large scale production and long term storage. Affibody molecules have also been engineered for extended circulation time to create molecules suitable for infrequent administration regimes. 

9:20 DARPins for Targeted Tumor Therapy and Diagnosis 
H. Kaspar Binz, Ph.D., Executive Director, Molecular Partners AG
Designed ankyrin repeat proteins (DARPins) are a novel class of highly stable binding proteins, which can be selected to bind to a broad spectrum of target proteins with high selectivity and affinity. We will show data on the engineering of multispecific DARPin variants binding to several targets at the same time without loosing their favorable properties. Also, we will present data on the excellent tumor accumulation and fast body clearance of radiolabeled DARPins in a xenograft mouse model. Our data suggest that DARPins are ideal candidates for targeted therapy and diagnosis in vivo.

9:50 Coffee Break in the Exhibit Hall

10:45 RNA-Mediated Chaperone as a Protein Folding Vehicle
Baik Seong, Ph.D., Professor, Department of Biotechnology, College of Engineering, Yonsei University, Korea
The expression of functionally active proteins in E.coli remains a formidable task despite extensive use of molecular chaperones or solubility-enhancing fusion carriers. Here, we report on a novel chaperone type of protein folding facilitated by interaction with RNA. The technology is by far superior to MBP-fusion and is extremely robust for soluble expression of a variety of proteins of human origin. This folding technology could be usefully implemented for high-throughput protein expression for functional and structural genomic research initiatives.

11:15 Structural Analysis of Human mAb Aggregates during Formulation Development 
Tiansheng Li, Ph.D., Preclinical Director, Pharmaceutics, Amgen Inc.
During the preclinical development of human monoclonal antibodies, aggregation and unfolding of monoclonal antibody are often observed. Due to the large molecular weight and complex structure of monoclonal antibodies, there are only limited biophysical techniques to study the structures of antibody aggregates and mis-folded antibodies. In this presentation, we shall discuss the use of vibrational spectroscopies including FT-IR, Vibrational Circular Dichroism (VCD), Raman and Raman Optical Activity (ROA), to investigate the structures of various types of monoclonal aggregates formed under different stresses.

11:45 TOSAP; A Novel Technology for Generating Protein Microspheres
Fang Fang, M.D., Ph.D., Chief Scientific Officer & Chief Medical Officer, NexBio Inc. 
TOSAP is a proprietary formulation technology for manufacture of microspheres suitable for pulmonary delivery. The process is robust and scaleable, uses no polymers and requires no specialized equipment. TOSAP platform was demonstrated to work on various classes of drugs including proteins and other molecules, with process yields close to 100%. The microspheres are loaded with 80 - 95% of the drug, have a narrow size distribution, excellent dispersivity and aerodynamic characteristics.

12:15pm Close of Morning Session

12:30 Luncheon Workshop 
or Lunch on Your Own 

1:45 Chairperson’s Remarks

1:50 Immunogenicity Assessment of Protein and Peptide Therapeutics
Philippe Stas, Ph.D., Chief Executive Officer, AlgoNomics NV
Most therapeutic proteins in clinical trials and on the market are to a variable extent immunogenic. Formation of anti-drug antibodies poses a risk that should be assessed during drug development, as it possibly compromises drug safety and alters drug characteristics including pharmacokinetics and bioavailability. The immunogenicity risk assessment is dependent on the nature of the protein therapeutic, and should be analysed on a case-to-case basis. This presentation reviews potential immunogenicity drivers, and their role in raising an immune response, and covers regulatory guidelines and fundamental techniques that can be used to assess the risk of immunogenicity in the different phases of protein drug development. 

2:20 TheraPEG-A Novel PEGylation Technology
Keith Powell, Ph.D., Chief Executive Officer, PolyTherics Ltd. 
Pegylation has been known for more than 20 years, but site-specificity on the protein has been problematic. Solutions such as inserting unnatural amino acids or terminal cysteine residues create their own problems. Now Polytherics has introduced TherapegTM, where addition of the PEG molecule at a disulphide bond gives site-specificity, a simple and scalable process, high efficiency and excellent half-life effects.

2:50 Development of an ACE2 Enzyme Substitution Therapy
Manfred Schuster, Ph.D., Chief Scientific Officer, Apeiron Biologics
Our most advanced project is the development of an Angiotensin Converting Enzyme 2 (ACE2) based enzyme substitution therapy to treat lung, kidney and heart diseases. We have established an industrial and scalable CHO-based production process to generate highly soluble, fully active, complex N-glycosylated and not immunogenic recombinant, human soluble ACE2. This purpose was achieved by a comprehensive clone selection strategy including productivity and product quality analysis at each selection round, including also intracellular FACS, activity and N glycosylation analysis. Systemic administration of ACE2 previously has confirmed its pharmacological properties and proven a therapeutic benefit in ARDS in an LPS induced sepsis model in piglets.

3:20 Technology Spotlight 

3:35 Refreshment Break in the Exhibit Hall

4:45 Poster Awards in the Exhibit Hall

5:00 Delivery Opportunities at the Interface of Peptides and Proteins
Christopher Rhodes, Ph.D., Senior Director, Drug Delivery Technology, Amylin Pharmaceuticals Inc.

5:30 Title to be Announced
Rick Costantino, Ph.D., Director, Formulations, Nastech Pharmaceutical Co. Inc.

6:00 End of Peptide & Protein-Based Therapeutics Conference