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Protect the healthy cells, expose the cancer - Transplant of engineered treatment-resistant Hematopoietic Stems Cells (eHSCs) to cure high-risk AML patients

May 31, 2023

Vortragsreihe "Cell and Gene Therapy"

Cell and gene therapies (CGTs) are a rapidly expanding sector of biopharmaceutical R&D, and these novel modalities are transforming how biopharma companies treat and potentially cure certain diseases. Cellular therapies include cellular immunotherapies, cancer vaccines, and other types of both autologous and allogeneic cells for certain therapeutic indications, including hematopoietic stem cells and adult and embryonic stem cells. Human gene therapies seek to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use. The development, manufacture, and distribution of CGTs are demanding, complex, resource intensive, expensive, and in many areas do not fit into the framework of the well-established processes for drug development and manufacture. Regulatory guidance is only now emerging, market access and reimbursement strategies need to be developed for CGTs that are curative but whose premium pricing is arguably taxing the healthcare system. The fast pace of innovation and the many uncertainties make CGT development a high-risk investment proposition. Many large pharma companies are investing in CGT, albeit cautiously, amid a nascent market. Most of today’s CGT innovation is still driven by small biotech companies that are either venture capital backed or must go through several rounds of private and public financing to fund their pipelines. 

Insights will be provided into CGT development at a small biotech start up in Cambridge, Massachusetts, one of the world’s largest and most vibrant biotech hubs. The company is built on a proprietary platform of hematopoietic stem cell biology, genome engineering, and CAR-T cells. The presentation will focus on key aspects of clinical development of treatment-resistant hematopoietic stems cells (eHSCs) as a curative treatment for patients with high-risk acute myeloid leukemia (AML). Further, the presentation aims to highlight some of the complexities of clinical trial execution, describe the product manufacturing process, and explain special aspects of cell therapy supply chain logistics.

Brief CV of the speaker:

Veit oversees the management of Vor Bio’s clinical research programs, working with cross-functional teams and external collaborators to advance the company’s engineered Hematopoietic Stem Cell (eHSC) platform. Veit has over 25 years of experience leading the developmentof novel therapies for cancer, autoimmune disorders, and infectious diseases at leading biopharmaceutical companies. Before joining Vor Bio, Veit was a Vice President at Magenta Therapeutics where he was project lead for MGTA-145, a novel biologic designed to mobilize HSCs prior to bone marrow transplant. Hepreviously served as Vice President of Portfolio Strategy at Mersana Therapeutics with responsibility for project leadership, alliance management,and portfolio strategy. Veit also served as Global Project Leader for Takeda Pharmaceuticals International (formerly Millennium Pharmaceuticals) where he was directly responsible for the company’s global asset strategy for new pipeline projects, overseeing programs in oncology from late-stage discovery through preclinical development and approval. Notably, Veit directed the global development of Entyvio® through Phase 2 and 3 clinical development, MAA/BLA review, global approvals, and launch. Veit began his career at Boehringer Ingelheim where he held positions of increasing seniority in his native Germany, the United States, and Japan.

Veit is a board-certified pharmacist in Germany where he obtained a PhD from Heidelberg University with a focus in formulation development.

Please register via Zoom.

We look forward to your participation.

Reinhold Roessler
Frank Kramer

FG Industriepharmazie
Veit Schelmer, PhD
Senior Vice President, Program and Alliance Management Vor Bio, Cambridge, MA, USA

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