On February 8, 2022 EdiGene, Inc., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious genetic diseases and cancer, reported a non-exclusive, worldwide license agreement with Arbor Biotechnologies, a biotechnology company discovering and developing the next generation of genetic medicines, to access Arbor’s proprietary CRISPR gene editing technology for certain ex vivo engineered cell therapy programs in the field of oncology (Press release, EdiGene, FEB 8, 2022, View Source [SID1234607865]).

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"Partnering with leading engineered cell therapy developers is a key component of Arbor’s strategy to broaden the potential of our CRISPR discovery engine beyond our wholly-owned in vivo genetic medicine approaches for the benefit of even more patients," said Devyn Smith, Ph.D., Chief Executive Officer of Arbor. "We look forward to expanding the global impact of Arbor’s proprietary gene editing technologies through our partnership with EdiGene."

The licensed technology is a part of Arbor’s proprietary CRISPR gene-editing portfolio, which is tailored to address the underlying pathology of genetic diseases.

"It has been great working with Arbor’s team in the past year to optimize ways of integrating Arbor’s proprietary CRISPR system into our ex vivo platforms," said Dong Wei, Ph.D., CEO of EdiGene. "This partnership will expand EdiGene’s gene-editing toolkit and strengthen our capabilities to advance ongoing ex vivo gene-editing cell therapy programs for multiple serious diseases. We look forward to collaborating with Arbor to achieve our shared commitment to developing innovative therapies to address unmet medical needs."

Under the agreement, EdiGene will pay Arbor certain upfront, milestone payments, commercial payments upon the achievement of certain development and sales milestones, and tiered royalties on net sales of royalty-bearing EdiGene products.

On February 8, 2022 Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on sickle cell disease, prostate cancer and other rare hematologic diseases and cancers, reported that it will present at the virtual 11th Annual SVB Leerink Global Healthcare Conference taking place Feb. 14-18, 2022 (Press release, Forma Therapeutics, FEB 8, 2022, View Source [SID1234607864]). Forma will present on Feb. 16, 2022 at 11:20 a.m. Eastern Standard Time (EST).

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A webcast of the conference presentation will be available in the "News & Investors" section of Forma’s website at www.FormaTherapeutics.com.

On February 8, 2022 Cyteir Therapeutics, Inc. ("Cyteir") (Nasdaq: CYT), a company focused on the discovery and development of next-generation synthetically lethal therapies for cancer, reported that the first patient has been dosed in a Phase 2 expansion cohort study of CYT-0851 monotherapy that is part of the Phase 1/2 trial. CYT-0851 is being evaluated for the treatment of hematologic malignancies and solid tumors (Press release, Cyteir Therapeutics, FEB 8, 2022, View Source [SID1234607863]).

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"We are excited to dose the first patient in the Phase 2 expansion cohort study with CYT-0851. This signal-seeking study in six different cancers will give us the data that may allow us to advance CYT-0851 into a clinical trial with potential registrational intent as soon as early next year," said Markus Renschler, MD, President and Chief Executive Officer of Cyteir. "I am grateful to the entire Cyteir team, our investigators and participating patients as we work together to complete these expansion cohorts."

CYT-0851 Phase 2 Monotherapy Study
CYT-0851 is a potent and selective, oral investigational drug that was designed to inhibit RAD51-mediated homologous recombination and the repair of double-strand DNA breaks. For the Phase 2 monotherapy expansion cohort study with CYT-0851 (NCT Number NCT03997968), we intend to enroll in six disease-specific cohorts in hematologic malignancies (relapsed and/or refractory diffuse large B-cell lymphoma, follicular lymphoma, and multiple myeloma) and solid tumors (recurrent metastatic or locally advanced pancreatic cancer, progressive ovarian cancer, and metastatic soft tissue sarcoma).

The Phase 2 study is being run with a Simon two-stage design and patients will be dosed with the recommended Phase 2 dose of CYT-0851 of 400 mg once daily that was determined in the Phase 1 dose escalation study. The objectives of the expansion cohort study are to evaluate preliminary anti-tumor activity in each disease specific expansion cohort, and to confirm the safety of the chosen Phase 2 dose.

On February 8, 2022 C2i Genomics, a cancer intelligence company, and Twist Bioscience Corporation (NASDAQ: TWST), a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, reported a partnership to develop whole-genome cancer reference materials (Press release, Twist Bioscience, FEB 8, 2022, View Source [SID1234607862]). This resource will provide diagnostic labs around the globe with the ability to better validate and monitor the quality of their whole-genome cancer screening and minimal residual disease (MRD) products. In addition, C2i will integrate Twist’s library preparation into their MRD workflow.

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Today in cancer diagnostics, a major obstacle facing diagnostic labs and regulators is the lack of standardized reference samples for labs to use for the analytical validation of their whole-genome assays. Especially in the complex field of oncology, there is a heightened need for advanced technology to help execute better treatment decisions, and ultimately, to save more lives.

Under the collaboration, C2i will use its artificial intelligence (AI) to identify a very large set of variants across the entire genome known to be associated with cancer signatures. Twist will then synthesize each of these variants, tuning them up or down to allow for similarity to natural samples, to create a whole-genome sequencing-focused set of reference materials. The reference standard kits will be available through Twist Bioscience.

"There is a tremendous need to have a consistent reference standard for the detection of cancer and minimal residual disease," said Emily M. Leproust, Ph.D., CEO and co-founder of Twist Bioscience. "We expect that the development of this exceptionally robust set of reference standards for whole-genome sequencing will complement our cfDNA Pan-Cancer Reference Standards, which can be used in liquid biopsy assays. We believe both of these products will enable our customers to create innovative solutions that accurately detect cancer initially and upon recurrence. C2i’s approach through AI and WGS has the potential to truly change the treatment paradigm."

The reference standards will also be used as training samples, tuning C2i’s algorithm for patient sample analysis. Using whole-genome sequencing and machine learning, C2i has developed a way to use (AI) to enable highly precise cancer detection in even the tiniest tumors. The development of this strategic collaboration demonstrates C2i’s investment in furthering the utility and accessibility to whole-genome cancer diagnostics in labs all over the world.

"C2i is committed to increasing the accessibility of whole-genome cancer detection and monitoring to support researchers and clinicians in improving oncology care for patients. We’re proud to work closely with Twist, as these new reference kits will solve an urgent unmet need for lab validation and proficiency testing materials in the cancer diagnosis space," said Asaf Zviran, CEO and Co-Founder at C2i Genomics.

C2i’s partnership with Twist comes after a year of significant momentum. C2i announced several key partnerships within the last year. The first being the initiation of its global commercial launch through its partnership with NovogeneAIT Genomics. This was followed by a network expansion with Premier Inc., a collaboration with NuProbe Global, and most recently an expansion into Europe with OncoDNA and the initiation of a first-of-its-kind interventional clinical trial in France led by Gustave Roussy. For companies interested in deploying the C2-Intelligence Platform, please visit www.c2i-genomics.com.

On February 8, 2022 Eureka Therapeutics, Inc., a clinical-stage biotechnology company developing novel T-cell therapies to treat solid tumors, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ET140203 and ECT204 for the treatment of hepatocellular carcinoma (HCC), the most common form of liver cancer (Press release, Eureka Therapeutics, FEB 8, 2022, View Source [SID1234607861]).

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Eureka is currently recruiting patients in three Phase I/II clinical trials to investigate the safety and potential efficacy of ARTEMIS T cells that have been engineered to target specific liver cancer antigens. The ARYA-1 and ARYA-2 studies use ET140203 ARTEMIS T cells to target an alpha fetoprotein (AFP)-peptide/HLA-A2 complex found on liver cancer cells. The ARYA-3 study uses ECT204 T cells to target the Glypican 3 (GPC3) protein expressed on the surface of liver cancer cells.

"We are pleased to have received ODD for ET140203 and ECT204 just months after receiving FDA Fast Track Designation (FTD) for the treatment of hepatoblastoma (HB) and HCC in pediatric patients, and Rare Pediatric Disease Designation (RPDD) for the treatment of HB," said Dr. Cheng Liu, President and CEO of Eureka Therapeutics. "These designations for Eureka’s clinical candidates underscore the significant unmet medical need for more effective liver cancer treatment options. We are committed to working closely with regulators, clinical investigators, patients and their families to advance these programs in the clinics."

Under the Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants and waiver of certain administrative fees for clinical trials, and seven years of market exclusivity following drug approval.

ABOUT ET140203

ET140203 is an investigational therapy during which a patient’s T cells are collected, engineered to express Eureka’s proprietary ARTEMIS cell receptor and infused back into the patient. Engineered ET140203 T cells express a TCR-mimic antibody to target an alpha fetoprotein (AFP)-peptide/HLA-A2 complex on liver cancer cells. In addition, ET140203 ARTEMIS T cells also incorporate Eureka’s proprietary tumor infiltration technology demonstrating enhanced ability to infiltrate solid tumors in animal models, potentially leading to improved efficacy in patients.

ET140203 is currently being investigated in two of Eureka’s ongoing clinical studies, ARYA-1 and ARYA-2: The ARYA-1 study is an open-label, dose escalation, multi-center Phase I/II clinical trial in adult patients with AFP-positive HCC; the ARYA-2 study is an open-label, dose escalation, multi-center Phase I/II clinical trial in pediatric subjects who are AFP-positive and have relapsed/refractory hepatoblastoma (HB), hepatocellular neoplasm not otherwise specified (HCN-NOS), and HCC.

ABOUT ECT204

ECT204 is an investigational ARTEMIS T-cell therapy targeting Glypican 3 (GPC3), a promising HCC antigen found in more than 70% of HCC cells. ECT204 ARTEMIS T cells also incorporate Eureka’s proprietary tumor infiltration technology demonstrating enhanced ability to infiltrate solid tumors in animal models, potentially leading to improved efficacy in patients. ECT204 is currently being investigated in Eureka’s ongoing ARYA-3 study, an open-label, dose escalation, multi-center Phase I/II clinical trial in adult patients with GPC3-positive HCC.

Additional information about the above studies may be found at www.clinicaltrials.gov, using Identifier NCT: NCT04502082, NCT04634357 and NCT04864054.