Update from the Clinic: February

Author: Mark Curtis, 03/24/16

Welcome to your Update from the Clinic for the month of February. bluebird made its debut in the oncology market and treated its first patient with a chimeric antigen receptor (CAR) product targeting a novel antigen. Ziopharm enrolled its first patient in a study investigating a non-viral CD19 CAR product, while Sangamo entered the clinic with an in vivo gene therapy for MPS I.

bluebird bio (BLUE) has been at the frontier of ex vivo gene therapy for rare blood disorders, including sickle cell and beta-thalassemia. The company has also been working away on a cell-based immunotherapy product (bb2121) in the background, partnered with Celgene, for patients with relapsed/refractory multiple myeloma. Unlike other liquid cancers, which have been heavily targeted by competitors such as Juno Therapeutics (JUNO) and Kite Pharma (KITE) particularly for acute lymphoblastic leukemia and non-Hodgkin’s lymphoma, multiple myeloma remains a space that is relatively niche. This, along with a unique target B cell maturation antigen (BCMA), gives bluebird a wedge into a competitive sector of oncology, and investors another reason to follow the story closely. Other notable companies going after multiple myeloma include Adaptimmune (ADAP) and Celyad (CYAD). Celgene has exercised its right to exclusively license bb2121.

Sangamo BioSciences (SGMO), which is developing a large and impressive pipeline of in vivo gene therapy products for rare disorders, announced FDA clearance of an investigational new drug application for its zinc finger nuclease (ZFN)-mediated treatment for mucopolysaccharidosis type I (MPS I). Like the first in vivo gene therapy product Sangamo brought into the clinic for hemophilia B (SB-FIX), the MPS I therapy (SB-318) is designed to leverage a patient’s liver as a factory for the production of therapeutic protein over the life of the patient. Current standard-of-care for rare disorders, such as MPS I, if any treatment exists, is typically enzyme replacement therapy, which is egregiously expensive, and requires bi-weekly injections over a patient’s life. The gene therapy space, in vivo and ex vivo, is one that is rapidly developing and becoming increasingly competitive, and Sangamo is positioning itself to be a formidable force.

CD19 has been a popular target in the clinic for both leukemias and lymphomas. Juno and Kite are both well into clinical development with CAR products directed to the antigen. Companies are differentiating themselves with novel approaches, like Juno’s “armored” CAR, which locally secretes signaling proteins to enhance immune response, and Cellectis’ (CLLS) universal cell product (UCART19) that leverages TALEN gene editing technology to cloak T cells from the host immune system. Ziopharm (ZIOP) is also in the mix, and enrolled its first patient this past month in a study of a CD19-specific CAR product for patients with lymphoid malignancies engineered using a non-viral approach with the Sleeping Beauty transposon-transpose system. Ziopharm expects a non-viral approach will deliver process efficiencies and drive down cost of manufacturing.

Disclaimer: “Update from the Clinic” is a blog post generated by news flow from public regenerative medicine (RM) companies around the globe. As CCRM has public RM companies in its industry consortium, and the number of such companies is relatively limited on a global scale, Mark Curtis will sometimes include CCRM consortium members in his review. This blog post is provided for general information only and nothing contained in the material constitutes a recommendation for the purchase or sale of any security. The author is not a shareholder of any public RM company. To see a list of CCRM’s industry consortium members, please visithttp://ccrm.ca/industry-consortium

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Mark Curtis

Mark Curtis

Mark is a Business Development Analyst at the Centre for Commercialization of Regenerative Medicine (CCRM), where he collaborates with the team to help evaluate the commercial potential of regenerative medicine and cell therapy technologies. He began his career at Princess Margaret Hospital studying cellular reprogramming of human skin cells. Following this project, he left the laboratory and took on a role with Bloom Burton & Co., a healthcare-focused investment dealer. While there he supported the advisory team in carrying out scientific diligence on early-stage biotechnology companies. Prior to joining CCRM he was a consultant to Stem Cell Therapeutics, a Toronto-based biotechnology company focused on developing therapeutics targeting cancer stem cells. Mark received a Master’s degree from the University of New South Wales in Sydney, where he studied the directed differentiation of embryonic stem cells, and a Bachelor’s degree in Biology, from Queen’s University. Follow Mark on Twitter @markallencurtis
Mark Curtis

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