Cambridge Healthtech Institute's 4th Annual

Fusion Protein Therapeutics

( 融合蛋白质疗法 )

Engineering Next-Generation Biologics

2018年4月30日-5月1日 | World Trade Center | 马萨诸塞州波士顿


The 4th annual Fusion Protein Therapeutics conference profiles the varying designs of therapeutic fusion proteins in differing stages of development, and investigates the challenges and benefits associated with these promising therapies. By combining modular building blocks that can reach targets not accessible to antibodies, Fusion Protein Therapeutics possess advantages over antibody-based therapies; their customizable functionality translates into lower patient dosing, reduced production costs, and improved product homogeneity. This conference will disclose how these molecules are being engineered to form more efficacious therapeutics that offer specificity with enhanced stability and longer half-life. Case studies from international experts will be presented covering R&D through clinical data.

Final Agenda

Sunday, April 30

Recommended Short Course(s)*

SC1: Preclinical and Clinical Assessment of Immunogenicity: Multidomain Therapeutics and New Modalities, Including Gene Therapy and CAR T

Darshana Jani, MS, Senior Manager, Pfizer, Inc.

Seema Kumar, PhD, Associate Director, Quantitative Pharmacology & Drug Disposition (QPD), R&D Global Early Development (GED), EMD Serono, a business of Merck KGaA, Darmstadt, Germany

Magdalena Tary-Lehmann, MD, PhD, CSO, Cellular Technology Limited (CTL); Adjunct Associate Professor of Pathology, Case Western Reserve University (CWRU)

Ravi Shankar Singh, PhD, Associate Director, Clinical Pharmacology, Pfizer

Ben Hock, PhD, Director, Immunogenicity, BioMarin Pharmaceuticals

*Separate registration required.


7:00 am Registration and Morning Coffee


8:30 Chairperson's Remarks

Stefan Weigand, PhD, Global Head, Large Molecule Research, Roche Diagnostics GmbH

8:40 Sotatercept, a Ligand Trap Fusion Protein, Attenuates Vascular Remodeling in Two Animal Models of Pulmonary Arterial Hypertension

Rovi_KumarRavindra Kumar, PhD, Senior Vice President & CSO, Acceleron Pharma, Inc.

Pulmonary arterial hypertension (PAH) is a progressive fatal disease characterized by remodeling of distal pulmonary arteries (more muscular), increased pulmonary vascular resistance, and right ventricular hypertrophy that ultimately lead to right heart failure. Mutations in BMP type II receptor (BMPR2) leading to reduced smad1/5/8 signaling have been implicated in familial PAH. Using in vitro and animal models of PAH, we show that sotatercept, a ligand trap based on soluble activin receptor type IIA, rebalances smad signaling and attenuates PAH.

9:10 A Novel Therapeutic Protein that Augments Tregs for the Treatment of Autoimmune and Inflammatory Diseases

Jeffrey_GreveJeffrey Greve, PhD, Founder and CSO, Delinia, Inc. (Celgene)

Regulatory T cells (Treg) play a major role in maintaining immune system homeostasis by regulating effector T cells and other immune cells. This homeostatic balance is lost in many autoimmune and inflammatory diseases. The IL-2 pathway is central to maintaining Treg levels and functional activity, and early-stage clinical trials in multiple autoimmune and inflammatory diseases have highlighted the potential of augmenting Tregs with low dose IL-2 to provide clinical benefit. However, there are significant significant toxicity and tolerability issues which severely limits the therapeutic use of IL-2. We have developed a novel Fc fusion protein that combines an IL-2 moiety that is highly selective for the IL-2 receptor expressed on Tregs with an Fc moiety to optimize circulating half-life. The development and characterization of this protein will be discussed.

9:40 Safety and Clinical Efficacy of AGT-181, a Brain Penetrating Human IgG-Iduronidase Fusion Protein, in a Phase 2 Study with Pediatric Patients with Mucopolysaccharidosis Type I

Ruben_BoadoRuben Boado, PhD, Vice President, Research & Discovery, ArmaGen Technologies, Inc.

AGT-181 is an IgG-enzyme fusion protein comprised of iduronidase (IDUA) and a monoclonal antibody against the human insulin receptor, engineered to cross the blood-brain-barrier (BBB) and to address both the neurocognitive and peripheral burden in MPS I. Neurocognitive function, somatic effects and safety of a phase II proof-of-concept clinical trial in Hurler MPSI pediatric patients will be discussed. This represents the first in human clinical trial of a fusion protein engineered to cross the BBB.

10:10 Networking Coffee Break


10:45 KEYNOTE PRESENTATION: Fc-Fusion Proteins: Past, Present and Future

Kenneth_WalkerKenneth W. Walker, PhD, Director, Research, Therapeutic Discovery, Amgen, Inc.

While there are many options for enhancing the pharmacokinetics of therapeutic proteins and peptides, Fc-fusion proteins are by far the most widely used. When designing Fc-fusion proteins, there are many factors to consider, which can interact in unexpected ways and have a substantial impact on the success of the molecule. In addition, some unconventional formats can substantially improve the manufacturability and/or activity of these molecules.

11:20 Characterization of Fusion Protein and Monoclonal Antibody Therapeutics during Drug Development - Techniques and Strategies

Li_TaoLi Tao, PhD, Research Fellow, Biologics Development, Bristol-Myers Squibb Company

The ever accelerated pace in fusion protein and antibody therapeutics development demands critical information to be generated and distributed to the key functional areas efficiently. This can only be achieved by state-of-the-art technologies and highly efficient business process. This presentation will focus on cutting edge analytical technologies and corresponding business process in support of protein therapeutics development to facilitate fast completion of different development phases. A few case studies will be presented.

11:50 Novel Reversible Switch for Protein Bioconjugates

Sunny_ZhouSunny Zhou, PhD, Professor and Faculty Fellow, Chemistry and Barnett Institute, Northeastern University

We recently invented several site-specific methods to install novel switches that can be removed reversibly. One example is by light and native form can be restored - a process often referred to as photocaging. An additional advantage is that the payload, if any, can be released simultaneously. Our method and newly developed switches have broad applications such as biological probes, fusion protein, antibody drug conjugates (ADCs) and controlled drug release.

12:20 pm Sponsored Presentation (Opportunity Available)

12:50 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on your Own

1:50 Session Break

2:20 Problem-Solving Breakout Discussions

3:20 Networking Refreshment Break


4:00 Chairperson's Remarks

4:10 Challenges and Opportunities in Engineering Protein Biopharmaceuticals

K. Dane Wittrup, PhD, C.P. Dubbs Professor, Chemical Engineering and Biological Engineering; Associate Director, Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology (MIT)

Arthur C. Clarke's First Law posits that "When a distinguished but elderly scientist states that something is possible, he is almost certainly right. When he states that something is impossible, he is very probably wrong." Bearing this in mind, in this talk I will highlight areas of protein drug development that appear poised for breakthroughs in the coming decade or so.

4:55 The Next Generation of Cancer Immunotherapy: Targeting Myeloid Immune Checkpoints

Kipp Weiskopf, MD, PhD, Resident Physician, Internal Medicine, Brigham and Women's Hospital

Immune cells of the myeloid lineage hold tremendous potential as effectors of cancer immunotherapy. The CD47/SIRPα axis is a key molecular pathway that governs the interaction between myeloid cells and tumors. Therapies that target the interaction are effective across multiple preclinical models of cancer and are now under investigation in clinical trials. Further studies have revealed additional regulators of myeloid cell activation that can be exploited as myeloid immune checkpoints.

5:40 Welcome Reception in the Exhibit Hall with Poster Viewing

7:15 End of Day


8:00 am Registration and Morning Coffee


8:25 Chairperson's Remarks

Patrik Stromberg, PhD, MBA, Senior Director, Head, Biomedical Science & Portfolio Innovation, Swedish Orphan Biovitrum AB (SOBI)

8:30 Agonist Redirected Checkpoints for Cancer Immunotherapy

Taylor_SchreiberTaylor Schreiber, MD, PhD, CSO and Co-Founder, Shattuck Labs, Inc.

Our groundbreaking immunotherapy harnesses the human immune system to improve survival for patients battling cancer and inflammatory disease. Agonist Redirected Checkpoint (ARC) technology allows creation of combination immunotherapy with a single product. In this talk, I will describe how we are using the ARC technology to develop next-generation immuno-oncology treatments.

9:00 Cell-Penetrating Peptides and TLR Peptide Agonist: The Swiss Army Knife of Cancer Vaccines

Madiha_DerouaziMadiha Derouazi, PhD, CEO, Amal Therapeutics

Modulating the immune system to enhance immune responses has become a promising therapeutic approach in oncology. We have developed a protein based therapeutic cancer vaccine platform called KISIMA. The technology is based on the assembly within one chimeric fusion protein of the following three elements: a cell penetrating peptide for antigen delivery, a TLR-peptide agonist as adjuvant and a modulable multi-antigenic cargo that can be tailored for various indications.

9:30 ImmTACs - Immuno-Oncology through TCR Targeted T Cell Redirection

Chris_CoughlinChristina M. Coughlin, MD, PhD, CMO, Immunocore, LLC

10:00 Coffee Break in the Exhibit Hall with Poster Viewing

10:50 Ontak-Like Human IL-2 Fusion Toxin

Zhirui_WangZhirui Wang, PhD, Assistant Professor, Surgery, Harvard Medical School; Senior Investigator/Head, Immunotoxin Laboratory, Center for Transplantation Sciences (CTS), Massachusetts General Hospital

Ontak® is an FDA approved diphtheria toxin-based fusion toxin for treatment of human CD25+ cutaneous T cell lymphoma (CTCL). However, it has been discontinued due to the production problem related to the bacterial expression system. Recently we have developed human IL-2 fusion toxin using advanced unique diphtheria toxin resistant yeast Pichia pastoris expression system. The in vivo efficacy was characterized using human CD25+ HUT102/6TG tumor-bearing immunodeficient NSG mouse model.

11:20 Development of a Novel Interleukin-2 Variant for Immunotherapy

Ekkehard_MoessnerEkkehard Moessner, PhD, Group Leader and Scientist, Protein Engineering, Roche Innovation Center Zurich

The development of an interleukin-2 mutein throughout the preclinical development will be described, until entering phase 1 in the clinic.

11:50 Optimizing IL2 Immunocytokines for Different Therapeutic Approaches - Simple Cytokine Targeting or Combined Treatment Modalities

Stephen_GilliesStephen D. Gillies, PhD, Founder & CEO, Provenance Biopharmaceuticals

Immunocytokines target cytokines with anti-tumor activity to the tumor microenvironment. This can mean fusing a cytokine to a tumor-specific scFv or instead, to a whole antibody containing binding sites for Fc receptors and C1q of the complement cascade. New approaches where the antibody component of the IC is an antagonist make it necessary to lower the cytokine bioactivity to match the concentration needed for inhibition of the target and avoid cytokine-induced toxicity.

12:20 pm Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on your Own

1:20 Ice Cream Break in the Exhibit Hall with Poster Viewing


2:00 Chairperson's Remarks

Sunny Zhou, PhD, Professor and Faculty Fellow, Chemistry and Barnett Institute, Northeastern University

2:05 ELVERA - A Novel Fully Human Fusion Protein Platform to Improve Drug-Like Properties of Biopharmaceuticals

Patrik_StrombergPatrik Stromberg, PhD, MBA, Senior Director, Head, Biomedical Science & Portfolio Innovation, Swedish Orphan Biovitrum AB (SOBI)

We are developing an innovative fusion protein technology that exploits the characteristics of a naturally occurring human protein sequence to protect and extend the circulatory half-life and target tissue exposure of active biological molecules. Using this technology, we were able to fine-tune the pharmacokinetic properties of therapeutic proteins, Affibody® molecules and antibody fragments. Also, we could show improved stability and reduced unspecific clearance of therapeutic enzymes. These results indicate that the ELVERA™ fusion protein technology could be used as a versatile platform to enhance the properties of a broad range of therapeutic proteins.

2:35 Nanofitin-Antibody Fusion as a Novel Multispecific Platform

Mathieu_CinierMathieu Cinier, PhD, CSO, Affilogic

Building on antibody expertise, many different bispecific formats have been engineered to allow the additional neutralization of an escaping pathway or the recruitment of effectors, but often at the expense of the overall physico-chemical properties of the biologic itself compared to the actual state of the art for monoclonal antibodies. Nanofitin-Antibody fusion is proposed as a novel multispecific platform, whereby additional targeting specificities are provided by the fusion of Nanofitins to existing antibodies without altering their initial physico-chemical properties.

3:05 Sponsored Presentation (Opportunity Available)

3:35 Refreshment Break in the Exhibit Hall with Poster Viewing

4:25 Multivalent Antibody-TRAIL Fusion Proteins for Cancer Therapy

Oliver_SeifertOliver Siefert, PhD, Scientist, Institute of Cell Biology and Immunology, University of Stuttgart

Engineering of multivalent antibody-scTRAIL (single-chain derivatives of TRAIL) by introducing different homodimerization modules leads to a novel platform of therapeutic molecules for cancer therapy. Our results show that both tumor targeting and enhancing the valency of scTRAIL fusion protein provides enforced apoptosis induction together with good anti-tumoral activity and tolerance in vivo. Due to the modular composition of this novel platform, exchanging the specificity of the antibody moiety facilitates the treatment of a broad spectrum of different cancer entities.

4:55 Fusion Proteins as an Efficient Drug Delivery Platform in Coagulation Therapy

Nicola_PozziNicola Pozzi, PhD, Assistant Professor, Biochemistry and Molecular Biology, Saint Louis University

Uncontrolled or inadequate delivery of current anticoagulant agents is the cause of bleeding diathesis and, in some cases, death. Engineering of fusion proteins is an attractive way to develop novel biotherapeutics with reduced side effects or enhanced pharmacokinetics. One such example is the rational design of anticoagulant fusion proteins obtained by fusing thrombin with the extracellular EGF456 domains of thrombomodulin through a peptide linker.

5:25 End of Fusion Protein Therapeutics

5:30 Registration for Dinner Short Courses

Recommended Dinner Short Course(s)*

SC13: Sub Visible Protein Particles in Immunogenicity: Measurement, Characterization and Impact

Bjorn Boll, PhD, Head, Particle Lab and Higher Order Structure Protein Analytics, Physical Chemical Analytics, Novartis Pharma AG

Anacelia Rios Quiroz, PhD, Scientist, Group Leader Particle Lab, Pharma Technical Development Europe (Biologics) Analytics (PTDE-A), F. Hoffmann-La Roche Ltd.

*Separate registration required.

* 活动内容有可能不事先告知作更动及调整。

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