on January 9, 2023 Avenge Bio, Inc. ("Avenge"), an oncology-focused biotechnology company developing the LOCOcyte Immunotherapy platform for the precision administration of potent immune effector molecules to treat solid tumors, reported its dosing of the first patient in a First-in-Human Phase 1/2 clinical trial evaluating AVB-001 in relapsed refractory ovarian cancer (Press release, Avenge Bio, JAN 9, 2023, View Source [SID1234626129]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

AVB-001, developed in the LOCOcyte platform, consists of proprietary engineered allogeneic human cells. The cells are encapsulated in a pro-inflammatory biomaterial that are delivered to the local tumor environment and generate high, sustained concentrations of native IL-2. The product initiates a robust and durable, local and systemic immune response while avoiding toxicities associated with systemic immunotherapies.

This first-in-human, single-arm, open-label, dose-escalation and expansion study (NCT05538624) is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of AVB-001 delivered intraperitoneally (IP) to patients with high grade serous adenocarcinoma of the ovary, primary peritoneum, or fallopian tube.

"The initiation of our first clinical trial of AVB-001 is a significant milestone for Avenge and the first candidate leveraging our LOCOcyte immunotherapy platform to enter the clinic. We are excited to advance AVB-001 as a potential treatment for patients with relapsed refractory ovarian cancer which has limited treatment options," said Michael Heffernan, Chief Executive Officer of Avenge.

"Ovarian cancer is one of the most difficult cancers to treat. It is typically not detected until later stages, and about 70 percent of patients will have recurrence after an initial treatment, which is often fatal. Immune checkpoint inhibitors have limited activity in this disease and there is a critical need for novel and effective therapies. Patients with ovarian cancer and other peritoneal malignancies are uniquely positioned to benefit from this novel cellular therapy," added Dr. Claudio Dansky Ullmann, Avenge’s Chief Medical Officer.

About LOCOcyte Platform
Our LOCOcyte allogeneic cell-based immunotherapy platform enables potent localized modulation of the immune system which also precipitates a systemic immune response, allowing us to treat previously intractable cancers. The technology leverage three unique advantages:

(1) Potent immune effector molecules are generated by synthetically engineering allogeneic cells creating a ready-to-use therapy,

(2) Therapy is localized in proximity to the primary tumor site and generates innate and adaptive immune response, and

(3) The immunomodulator trains the patient’s immune system generating a robust immune response that seeks and eradicates distal metastasis without systemic toxicity.

On January 9, 2023 Illumina Inc. (NASDAQ: ILMN), a global leader in DNA sequencing and array-based technologies, and Nashville Biosciences LLC, a wholly owned subsidiary of Vanderbilt University Medical Center (VUMC), reported an agreement with Amgen, a global biopharmaceutical company, to whole-genome sequence approximately 35,000 DNA samples (Press release, Illumina, JAN 9, 2023, View Source [SID1234626128]). The sample cohort is primarily made up of DNA from African Americans, who are currently underrepresented in research for the clinical applications of genomics, including drug target discovery. This cohort will be the largest data set of genomes of its kind to date.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Illumina and Nashville Biosciences reported an agreement with Amgen to whole-genome sequence approximately 35,000 DNA samples. The sample cohort is primarily made up of DNA from African Americans, who are currently underrepresented in research for the clinical applications of genomics, including drug target discovery. Whole-genome sequencing will be performed by deCODE genetics, a wholly owned subsidiary of Amgen. Image courtesy of deCODE genetics.
Illumina and Nashville Biosciences reported an agreement with Amgen to whole-genome sequence approximately 35,000 DNA samples. The sample cohort is primarily made up of DNA from African Americans, who are currently underrepresented in research for the clinical applications of genomics, including drug target discovery. Whole-genome sequencing will be performed by deCODE genetics, a wholly owned subsidiary of Amgen. Image courtesy of deCODE genetics.
It’s widely recognized that most genomic data sets are drawn from people of European ancestry. This lack of diversity in genomic data has created a gap in the scientific understanding of the underlying genetic causes of disease and inhibits equitable access to precision health therapies.

Sequencing this set of samples is the first in Illumina and Nashville Biosciences’ Alliance for Genomic Discovery (AGD), a multiyear agreement to accelerate therapeutic development through large-scale genomics and establish a preeminent clinico-genomic data set.

Launched in 2022, the AGD aims to whole-genome sequence at least 250,000 de-identified human DNA samples from VUMC’s BioVU biobank over two and a half years in collaboration with multiple biopharmaceutical companies. The BioVU samples were extracted from blood collected during routine clinical testing. The patients who provided them consented to research use, and the samples are linked to extensive de-identified clinical data derived from VUMC’s electronic medical records.

"The whole-genome sequencing of these 35,000 samples will work toward greater diversity of genomic data to ultimately enable improved access to precision therapies for all people," said Joydeep Goswami, chief strategy and corporate development officer and interim chief financial officer of Illumina.

"The initial cohort will be among the largest sequencing efforts involving African Americans to date," said Leeland Ekstrom, chief executive officer of Nashville Biosciences. "Once complete, this data set will provide a wealth of new information about the human genome and accelerate the study of disease in—and discovery of new therapeutics for—populations less well represented in prior large-scale sequencing efforts. The opportunity to sequence this diverse set of samples will help broaden our understanding for individuals who have been underrepresented in genetic research and continue to experience health disparities."

As part of the agreement announced today, deCODE genetics, a wholly owned subsidiary of Amgen, will perform whole-genome sequencing on the 35,000 samples using Illumina sequencing technologies and will upload the data to the Illumina Connected Analytics platform.

"deCODE’s sophisticated human data capabilities are well positioned to sequence these samples and analyze the resulting data to provide a better scientific understanding of disease through the context of human diversity," said Kári Stefánsson, founder of deCODE genetics. "Insights from human data, including both genetics and clinical records, can help inform how medicines may affect different patient populations."

Pharma and biotech collaborators that plan to participate in the AGD and in further sequencing may soon be announced. Researchers will analyze the data for drug discovery and therapy development. The data will also be returned to BioVU to be made available for academic research within the Vanderbilt community.

On January 9, 2023 Secura Bio, Inc. (Secura Bio) – (www.securabio.com), an integrated pharmaceutical company dedicated to the worldwide development and commercialization of impactful oncology therapies, reported that the European Commission (EC) issued an Orphan Drug Designation for duvelisib for the treatment of patients with peripheral T-cell lymphoma (PTCL) (Press release, Secura Bio, JAN 9, 2023, View Source [SID1234626127]). Duvelisib was previously granted orphan drug designation by the United States Food and Drug Administration.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Orphan Drug Designation in the EU is granted by the EC based on a positive opinion issued by the European Medicines Agency Committee for Orphan Medicinal Products. It is intended to encourage the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases. If approved for marketing, this designation will provide ten years of marketing exclusivity and other special incentives for sponsors, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees.

Duvelisib is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first US-approved dual inhibitor of PI3K-delta and gamma pathways, which are involved in the proliferation and sustenance of malignant cells. Duvelisib was fully approved by the US Food and Drug Administration in September 2018 for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma after at least two prior therapies.

"This Orphan Drug Designation recognizes the significant unmet need in patients with PTCL, especially those with relapsed/refractory disease. This patient group has very limited therapeutic options and duvelisib may offer a new choice of therapy." Said Dr. David Sidransky, Clinical Advisor to Secura Bio.

"Secura Bio is dedicated to developing duvelisib for the treatment of patients with difficult-to-treat cancers, which includes relapsed/refractory PTCL. We are investing significant corporate resources in this endeavor and hope to see new treatment options brought to the market which may benefit patients, such as those with relapse/refractory PTCL." Said Joseph M. Limber, President and CEO of Secura Bio.

On January 9, 2023 Bantam Pharmaceutical, LLC reported that it is presenting at the Biotech Showcase taking place in San Francisco, the 9th-11th of January and virtually, on demand the 18th and 19th of January (Press release, Bantam Pharmaceutical, JAN 9, 2023, View Source [SID1234626125]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Bantam Pharmaceutical is developing first-in-class therapeutics that leverage the "cellular power plants," known as mitochondria, to destroy cancer cells.

Bantam has a novel, oral therapeutic, BTM-3566, with an FDA-cleared IND featuring a phase 1 study in B cell lymphomas. BTM-3566 targets mitochondrial homeostasis, with exceptional single-agent activity in hematological malignancies, including complete responses in diffuse large B-Cell lymphoma (DLBCL) in vivo models. These models were derived from patient tumors with multiple genomic alterations that underlie the high death rates in these difficult-to-treat lymphomas.

The presentation by Bantam Pharmaceutical President and CEO Michael Stocum will cover BTM-3566 novel anti-cancer mechanism and the company’s potential to bring novel cancer agents closer to patients in need. Bantam features a leadership team of industry veterans, with most having over two decades of experience across several therapeutic areas in pharmaceuticals, biologics, and molecular diagnostics.

On January 9, 2023 Bio-Techne Corporation (NASDAQ: TECH) reported the expansion of its Advanced Cell Diagnostics (ACD)-branded RNAscope in situ hybridization (ISH) portfolio with the release of the RNAscope Plus smRNA-RNA detection assay (Press release, Bio-Techne, JAN 9, 2023, View Source [SID1234626124]). The RNAscope Plus smRNA-RNA assay enables the simultaneous fluorescent detection of a small regulatory RNA together with 3 target RNAs or RNA biomarkers in the same tissue section at single-cell and subcellular resolution. Designed to be easily scalable to support large studies, the assay will be offered for use on the Leica Bond Rx platform and as a manual assay.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Small regulatory RNAs, including miRNAs, are instrumental in regulating human health and have been associated with a wide range of different diseases. Oligonucleotide therapies, including antisense oligonucleotides (ASOs) and RNA interference (RNAi), represent a unique class of gene therapies that have the potential to provide highly focused, long-lasting therapeutic effects by utilizing similar cellular processes. These therapeutic nucleic acids can be used to regulate the expression and function of specific target genes, enabling the development of tailored therapies for inherited or acquired diseases that have been previously resistant to standard therapies.

Advanced Cell Diagnostics miRNAscope ISH technology has become the gold standard for identifying the cellular localization of endogenous miRNAs and measuring delivery efficiency of therapeutic oligonucleotides in preclinical animal model systems. Combining proprietary RNAscope and miRNAscope ISH technologies, the RNAscope Plus smRNA-RNA detection assay provides researchers with a valuable new tool to quantify changes in gene expression and cellular function in response to the introduction of regulatory RNAs, essential for optimization of therapeutic efficacy and safety.

"We are excited to further expand our unique offering for the gene therapy community with the launch of the RNAscope Plus smRNA-RNA detection assay. We received an overwhelmingly positive customer response to this novel assay following introduction by our Professional Assay Services and are thrilled to provide broader access to accelerate the groundbreaking work of our customers," said Kim Kelderman, President of Bio-Techne’s Diagnostics and Genomics Segment.