CELL AND GENE THERAPY

BioInvent and Transgene Receive CTA Approval for Phase l/lla Study of Novel Oncolytic Vaccinia Virus BT-001

BioInvent | December 22, 2020

BioInvent International AB ("BioInvent") (OMXS: BINV), a biotech organization focused in on the discovery and development of novel and first-in-class immune-modulatory antibodies for cancer immunotherapy, and Transgene (Euronext Paris: TNG), a biotech organization that designs and develops infection based for the therapy of cancer, reported they have received administrative endorsement in Belgium for a clinical trial application (CTA) for a Phase l/lla study of the novel oncolytic vaccinia virus BT-001.

BT-001 is a best-in-class oncolytic Vaccinia virus. It has been produced utilizing Transgene's Invir.IO™ stage and its protected large-capacity VVcopTK-RR-oncolytic virus, which has been designed to encode both a Treg- depleting human recombinant anti-CTLA4 antibody response created by BioInvent's proprietary n-CoDeR®/F.I.R.S.T™ stages, and the human GM-CSF cytokine. By specifically focusing on the tumor microenvironment, BT-001 is relied upon to inspire a lot more grounded and more successful antitumoral reaction. Conveying the anti-CTLA4 antibody response straightforwardly to the tumor microenvironment will permit a neighborhood remedial movement and will in this manner enormously increment the security and tolerability profile of the monoclonal immunizer by antibody foundational introduction. BT-001 is being co-created through a 50/50 collaboration among BioInvent and Transgene.

Spotlight

The first biosimilar approved under the abbreviated pathway established by the Biologics Price Competition and Innovation Act launched in the U.S. in 2015.

Spotlight

The first biosimilar approved under the abbreviated pathway established by the Biologics Price Competition and Innovation Act launched in the U.S. in 2015.

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MEDTECH, INDUSTRIAL IMPACT

SOPHiA GENETICS and QIAGEN Partner to Boost Next-gen Sequencing

SOPHiA GENETICS and QIAGEN | March 06, 2023

SOPHiA GENETICS, a healthcare cloud-based software company, announced a new partnership with QIAGEN N.V. that will pair QIAseq reagent technology with the SOPHiA DDM™ platform to improve tumor analysis via next-generation sequencing (NGS). Customers can utilize QIAseq panels with SOPHiA DDM™, a cloud-based data analysis platform to enable new research perspectives. The partnership will support the detection of somatic variants using QIAseq targeted DNA pro panels for homologous recombination repair. HRR is a biomarker test that helps identify tumors with specific mutations that may be sensitive to PARP inhibitors in oncology. SOPHiA GENETICS's Set-Up Program, which aims to establish and demonstrate any test's analytical performance before it is carried out, will be integrated with QIAGEN's kits to support a range of applications. This partnership will allow customers to improve the design of new workflows using QIAseq technologies, while SOPHiA DDM™ will offer extensive applications for QIAGEN kits. QIAGEN intends to expand its partnership with SOPHiA GENETICS to other analysis areas, such as utilizing custom solutions and QIAseq panels. This partnership is the first one in QIAGEN's QIAseq Platform Partnership. Next-generation sequencing (NGS) can transform the detection and management of rare diseases and cancers. However, it also generates large, intricate, and messy datasets that are challenging to analyze. Identifying causal variants and getting valuable insights from NGS data requires specialized expertise, time, and numerous resources. The SOPHiA DDM™ Platform is a machine learning-based analytical tool that can efficiently call, annotate, and pre-classify variants from raw NGS data. In addition, it features user-friendly capabilities to facilitate prioritization, interpretation and reporting. About QIAGEN QIAGEN is a global provider of sample and assay technologies for academic and pharmaceutical research, molecular diagnostics, and applied tests. In addition, the firm offers a wide range of products and solutions for the isolation, purification, and analysis of DNA, RNA, and proteins, as well as automation and bioinformatics services. With over 35 countries and over 5,000 employees, QIAGEN serves customers in various fields such as human healthcare, animal health, and environmental testing. The company is headquartered in Venlo, Netherlands, and has offices and facilities across the globe.

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CELL AND GENE THERAPY, INDUSTRIAL IMPACT

Exelixis and Sairopa Announce FDA Clearance of IND Application

Exelixis | February 14, 2023

On February 13, 2023, Exelixis, Inc., a leading global oncology firm innovating next-generation medicines and regimens at the forefront of cancer care and Sairopa B.V. (Sairopa), a renowned firm developing novel therapeutics for cancer by modulating the patient's immune system, announced that FDA has approved Sairopa's Investigational New Drug (IND) Application to assess the pharmacokinetics and safety of ADU-1805 in adults with advanced solid tumors. ADU-1805, being a monoclonal antibody active against all human SIRPα alleles, has the potential to reach a broader patient population than existing SIRPα-directed therapeutics. ADU-1805 has the potential to boost the immune system's ability to combat tumors by suppressing SIRPα, an important immuno-suppressive component of the tumor microenvironment. When compared to other SIRP family members, ADU-1805 has been designed to bind preferentially to SIRPα, which may improve its capacity to stimulate immune cells. In accordance with the clinical development and option agreement announced in November 2022, Exelixis has the option to obtain an exclusive, global license to commercialize and develop ADU-1805 and other anti-SIRPα antibodies after reviewing data from predetermined phase 1 clinical studies of ADU-1805 to be conducted by Sairopa during the option period. This IND approval results in a $35 million milestone payment to Sairopa to be paid in the first quarter of 2023. About Exelixis Founded in 1994, Exelixis is a leading global oncology firm at the vanguard of cancer care, developing next-generation drugs and regimens. Fueled by bicoastal centers of research and development expertise, its clinically distinct pipeline of small molecules, antibody-drug conjugates, and other biotherapeutics is quickly expanding its product portfolio to address a growing spectrum of tumor types and indications. In addition, the company has demonstrated an excellent track record of endurance in the face of adversity after working in the competitive biotech industry for over 25 years. Its main product's success has provided it with a robust commercial basis, allowing re-energizing research activities and team expansion in clinical development, drug discovery, and commercial areas.

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CELL AND GENE THERAPY, INDUSTRIAL IMPACT

Lineage Enters Exclusive Option and License Agreement with Eterna

Lineage Cell Therapeutics, Inc. | February 23, 2023

On February 22, 2023, Lineage Cell Therapeutics, Inc., a clinical-stage biotechnology firm focused on the development of allogeneic cell therapies to restore and replace specific cell types of the human body, announced that it has entered into an exclusive option and license agreement with Eterna Therapeutics Inc. (Eterna) for the development of innovative beta 2 microglobulin (B2M)-deficient induced pluripotent stem cell (iPSC) lines that Lineage will assess for the creation of unique, differentiated cell transplant therapies. These new cell lines to be developed by Eterna are expected to help Lineage generate more product candidates for the treatment of central nervous system (CNS) conditions and other neurology indications. Eterna holds the exclusive license for the crucial intellectual property associated with this partnership, which it obtained from its discovery partner Factor Bioscience. Following the agreement, Eterna intends to perform specific gene-editing activities and furnish materials to Lineage for assessment. In addition, the agreement grants Lineage the option to obtain an exclusive license to use and sublicense the new gene-edited cell lines for clinical, preclinical, and commercial purposes in the field of CNS diseases. A distinctive feature of the starting cell line is the targeted elimination of the B2M gene, which is aimed at minimizing the immunogenicity of product candidates generated from the cell lines by inhibiting rejection by CD8+ T cells. Lineage anticipates that this attribute will enhance the overall usefulness of the edited cell lines, including non-immune privileged or non-human leukocyte antigen (HLA) matched indications. Other proposed gene modifications may additionally differentiate the cell line from those currently utilized by competitors. No details of the financial agreement were disclosed. About Lineage Cell Therapeutics, Inc. Founded in 1990, Lineage Cell Therapeutics is a clinical-stage biotechnology company focused on developing novel cell therapies to address unmet medical needs. The company's programs are based on its proprietary cell-based therapy platform and related development and manufacturing capabilities. By using this platform, it specializes in the development and manufacture of specialized, terminally-differentiated human cells from its progenitor and pluripotent cell starting materials. These differentiated cells are intended to either support or replace cells that are dysfunctional or absent due to degenerative disease or traumatic injury and can also be administered to help the body mount an effective immune response to cancer.

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