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• Phase 1b study includes Parkinson’s disease patients with and without a genetic LRRK2 mutation

SOUTH SAN FRANCISCO, Calif., Dec. 10, 2018 (GLOBE NEWSWIRE) — Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates for neurodegenerative diseases, today announced initiation of dosing in a Phase 1b clinical study of DNL201 in patients with Parkinson’s disease.

“Based on the positive outcome of our Phase 1 study in 122 healthy volunteer subjects, we are excited to evaluate DNL201 in Parkinson’s disease patients,” said Carole Ho, M.D., Chief Medical Officer of Denali. “This study will provide additional important safety and biomarker data in patients to support rational dose selection. The results from this study will inform decisions on further clinical testing of DNL201, including potential registrational trials.”

DNL201, Denali’s lead LRRK2 therapeutic candidate, is a small molecule inhibitor of leucine-rich repeat kinase 2 (LRRK2). LRRK2 is a regulator of lysosomal function, which is impaired in Parkinson’s disease and may be restored by LRRK2 inhibition. Inhibition of LRRK2 activity may potentially slow the progression of disease in patients with a genetic LRRK2 mutation as well as in patients with sporadic Parkinson’s disease.

Mutations in the LRRK2 gene are among the most frequent genetic causes of Parkinson’s disease and a major driver of lysosomal dysfunction, which contributes to the formation of Lewy body protein aggregates and neurodegeneration.

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SpaceX Falcon 9 Delivering ARCUS Gene Editing to the International Space Station

CAPE CANAVERAL, Fla.–(BUSINESS WIRE)–Precision BioSciences today announced that the world’s first genome editing experiment in space has been launched aboard a SpaceX Dragon cargo spacecraft on a Falcon 9 rocket. The mission was launched yesterday from Cape Canaveral Air Force Station and is anticipated to deliver its cargo to the International Space Station (ISS). Astronauts aboard the ISS are expected to perform the first genome editing experiment in space using Precision’s proprietary, next-generation genome editing technology, ARCUS.

Precisioneers JoAnn Hux and Ginger Tomberlin have been working with Karen Kingera, STEM Director of Immaculata Middle School in Durham, North Carolina, and her dedicated group of 15 students for over a year on this project. The program was made possible by Space Center University at Space Center Houston and the DreamUp program. DreamUp places select student projects on SpaceX ISS payloads, and students whose projects are chosen are also invited to attend the launch and participate in presentations and poster sessions at Cape Canaveral.

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Unum Therapeutics Inc. (NASDAQ: UMRX), announced today that the United States Patent and Trademark Office has issued US patent 10,144,770, entitled “Chimeric Receptors and Uses Thereof in Immune Therapy.” The ‘770 patent covers design and use of the chimeric Antibody-Coupled T-cell Receptor (ACTR) platform technology that enables an engineered immune cell to be targeted via an antibody to attack certain cell types, including cancer cells. An ACTR-expressing T cell offers a number of potential advantages over alternative cell therapy approaches and clinical proof-of-concept for the ACTR technology has been demonstrated in two independent clinical trials sponsored by Unum Therapeutics. ACTR therapeutic programs targeting non-Hodgkin lymphoma, multiple myeloma, and HER2+ advanced cancers are currently in early stages of clinical testing.

The initial ACTR construct was developed by Dr. Dario Campana, a well-renowned cancer researcher recognized as an inventor of the chimeric antigen receptor that forms the basis for tisagenlecleucel, the world’s first approved CAR-T therapy. Working initially at St. Jude Children’s Research Hospital and later at the National University of Singapore, Dr. Campana and his team were able to demonstrate in mouse studies that ACTR-expressing T cells exhibit potent anti-tumor activity. Researchers at Unum Therapeutics extended upon this work, demonstrating that a wide range of functional ACTR receptors could be constructed using different building blocks derived from various human immune cell receptors.

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First demonstration of potential gene therapy treatment in this population leading to sustained levels of functioning neutrophils after 12 months

BOSTON and LONDON, Dec. 05, 2018 (GLOBE NEWSWIRE) — Orchard Therapeutics (NASDAQ:ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that Scientific Advisory Board Member, Donald B. Kohn, MD, presented clinical proof-of-concept data from an ongoing academic clinical trial evaluating OTL-102 for the treatment of X-Linked Chronic Granulomatous Disease (X-CGD) during the Presidential Symposium at the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition on December 4, 2018. X-CGD is a life-threatening inherited immunodeficiency disorder which is caused by a genetic mutation that results in the inability of neutrophils to effectively kill bacterial and fungal pathogens. This immune deficiency leads to repeated chronic and severe infections often requiring hospitalization and resulting in chronic sequelae leading to early mortality. Preclinical and clinical development of OTL-102 had originally been initiated by Genethon (Evry, France) before licensing to Orchard.

“These clinical proof-of-concept data demonstrate, for the first time, 12 months or more of restoration of immunity in X-CGD patients treated with ex-vivo autologous gene therapy,” said Adrian Thrasher, professor of pediatric immunology and Wellcome Trust Principal Research Fellow at UCL Great Ormond Street Institute of Child Health in London. “In six of the seven evaluated patients, we are encouraged to see sustained levels of functioning neutrophils at greater than 10%, which prior publicly available data suggests is the level sufficient to see potential clinical benefit. This important milestone, together with improved clinical outcomes and an emerging safety profile with no signs of genotoxicity, suggest OTL-102 may provide a transformative treatment for X-CGD patients.”

SUZHOU, China, Dec. 4, 2018 /PRNewswire/ — Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class China-based biopharmaceutical company that develops and commercializes high quality drugs, announced today that its IND application for IBI101, a recombinant fully human anti-OX40 monoclonal antibody (mAb) drug candidate, has been approved by the US Food and Drug Administration (FDA), and plans to initiate the US phase I clinical trial based on results from the China phase I study in patients with advanced solid tumors.

IBI101 is the third molecule from Innovent approved for clinical trials by FDA. The company also has received FDA IND approvals for IBI308 (Sintilimab, an anti-PD-1 antibody) in January, 2018 and IBI188 (an anti-CD47 antibody) in September, 2018 respectively. Innovent is the first Chinese biopharmaceutical company to receive clinical trial approval from FDA for an anti-OX40 monoclonal antibody.

“Innovent was built according to international R&D and production standards and has been exploring the latest cutting-edge research in the area of biopharmaceutical development. The FDA IND approval of anti-OX40 monoclonal antibody once again demonstrates Innovent’s research and development capability. We hope that through our efforts, we can make more breakthroughs in the field of innovative biopharmaceuticals and benefit patients around the world,” said Michael Yu, Founder, Chief Executive Officer and Chairman.

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NEW YORK & OTTAWA, Ontario–(BUSINESS WIRE)–Turnstone Biologics, a clinical-stage immuno-oncology company leading the next generation of oncolytic viral therapies, presented pre-clinical data today supporting the development of a new Vaccinia therapeutic platform at the American Association for Cancer Research Tumor Immunology and Immunotherapy Meeting in Miami. This platform was developed by Dr. John Bell and his colleagues at The Ottawa Hospital Research Institute and the University of Ottawa, and engineered to be potent, highly selective and immune-stimulatory. Furthermore, the versatility of the platform enables additional therapeutic agents to be encoded in the virus and produced at tumor sites. This single platform virus has the potential to create a portfolio of diverse products. Turnstone has exclusively licensed this technology.

This proprietary Vaccinia virus can be delivered systemically and is designed to target and kill cancer cells throughout the body, modulate the tumor microenvironment and stimulate both innate and adaptive immune responses. The virus also contains a very large transgene capacity, allowing for the insertion of multiple therapeutic agents. These agents will be produced selectively at tumor sites as the virus replicates only in the tumor cells, effectively enabling a local combination therapy from a single therapeutic promoting cancer cell killing. Turnstone is conducting IND-enabling studies on TBio-6517, its lead product candidate from this platform, consisting of the Vaccinia virus expressing three potent immune modulators, and anticipates initiating clinical studies in 2019.

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Hutchison China MediTech Ltd. (LSE:HCM; NASDAQ:HCM) and Innovent Biologics Inc. (HKSE:1801) will take advantage of China’s new foreign data guidelines and enter into a global partnership to develop fruquintinib in combination with sintilimab to treat advanced solid tumors.

The companies said the new foreign data guidelines, which allow companies to submit foreign clinical data to support Chinese regulatory submissions, could expedite the path to a China launch (see “China Accelerates Drug Review Timelines for Rare, Life-Threatening Diseases”).

Innovent’s sintilimab, an anti-PD-1 mAb, is under review in China to treat relapsed or refractory classical Hodgkin lymphoma.

Elunate fruquintinib is approved in China to treat metastatic colorectal cancer (mCRC). According to Chi-Med, the inhibitor of VEGF receptors 1, 2 and 3 is the first China-discovered and developed product for an oncology indication to receive unconditional approval based on a randomized clinical trial (see “Chi-Med Scores a China First with Approval of Fruquintinib”).

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Global collaboration will initially evaluate Innovent’s anti-PD-1 monoclonal antibody sintilimab in combination with Hutchison MediPharma’s VEGFR inhibitor fruquintinib for solid tumors –

SUZHOU, China, Nov. 29, 2018 /PRNewswire/ — Innovent Biologics, Inc. (Innovent) (HKEX: 1801),  a world-class China-based biopharmaceutical company that develops and commercializes high quality drugs, today announces that it, through its wholly-owned subsidiary, Innovent Biologics (Suzhou) Co., Ltd, has entered into a global collaboration agreement with Hutchison China MediTech Limited (Chi-Med), through its Innovation Platform subsidiary Hutchison MediPharma Limited (“Hutchison MediPharma”), to evaluate the safety and tolerability of Innovent’s sintilimab in combination with Hutchison MediPharma’s fruquintinib in patients with advanced solid tumors.

Under the terms of the agreement, Innovent and Hutchison MediPharma will jointly explore potential application of this combination in solid tumors with global unmet medical needs through development efforts both in the US and in China.

“We are two leading China-based biopharmaceutical companies, one specialized in small molecules and another in large molecules; and we share the same vision of bringing China-originated mainstream anti-cancer therapies to global patients by combining our expertise and resources,” said Dr. Michael Yu, Founder, Chief Executive Officer and Chairman of Innovent. “There is strong scientific evidence supporting synergistic effects of PD-1 therapy when used in combination with VEGFR inhibitor. In addition, we hope to benefit from recent regulatory changes in China that allow for the recognition of foreign clinical trial data to possibly expedite the path to a China launch. We are very pleased to partner with Chi-Med to co-develop this novel combination therapy for global patients”.

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