On April 2, 2024 Takara Bio USA, Inc., a wholly owned subsidiary of Takara Bio Inc., reported the launch of the Shasta Single-Cell System, an automated, high-throughput NGS solution with well-validated chemistries and intuitive bioinformatics tools that enables novel biomarker discovery for oncology research (Press release, Takara Bio, APR 2, 2024, View Source [SID1234641728]). This complete system allows researchers to mine more genomic and transcriptomic information from many more cells than possible with current technologies, while saving time and costs for research groups. Existing whole-genome amplification (WGA) technologies currently process 96–384 single cells per plate; the Shasta system increases WGA throughput to 1,500 cells per run. With its total RNA-seq application, the Shasta system detects more RNA biotypes with high sensitivity at high throughput—up to 100,000 cells per run—which is an improvement over both plate-based full-length RNA-seq and high-throughput mRNA-seq methods.

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In March, Takara Bio USA delivered instruments and NGS kits to early-access users, who have started producing results with the system. The company is accepting orders with plans to ship instruments and chemistries towards the end of Q2. "We’re very excited about this technology," said early-access user Dr. Ting Wang, Head, Department of Genetics at Washington University School of Medicine in St. Louis. "I want to expand our usage of this technology. We have two other projects in the lab where we want to do a full set of profiling of our entire model system. We’re fully committed to continue pushing the limit of the technology to do the best science."

The Shasta system has three core elements: a state-of-the-art, automated dispensing and imaging platform, a unique array of NGS library prep chemistries, and Cogent bioinformatics software for comprehensive data analysis—easy to use even for researchers and staff without bioinformatics experience.

"We have reached a new era in single-cell analysis. Researchers are no longer limited to profiling just hundreds of cells or accessing only part of a cell’s rich genetic information. Shasta technologies have broken the limits of current methods to allow the detection of events like splicing isoforms, gene fusions, long noncoding RNAs, and arm-level CNVs—without sacrificing sensitivity or scale," said Carol Lou, President & CEO of Takara Bio USA. Researchers can take advantage of three applications:

The Shasta Total RNA-Seq Kit profiles the full-length transcriptomes of up to 100,000 single cells and up to 96 samples per experiment with only two rounds of barcoding. The random-priming-based chemistry enables the discovery of novel types of biomarkers beyond mRNA, facilitating understanding of disease mechanisms and therapeutic responses.
The Shasta Whole-Genome Amplification Kit features a fully automated protocol that prepares single-cell DNA-seq libraries for up to 8 samples and 1,500 single cells per run, reducing hands-on time. This application enables researchers to scale up their WGA workflow to understand tumor heterogeneity and perform tumor subclonal analysis through copy number variation (CNV) and single nucleotide variation (SNV) profiling at a shallower sequencing depth, saving costs.
The Shasta mRNA-Seq Kit accomplishes full-length transcript coverage with outstanding sensitivity for up to 1,500 single cells per run. This application delivers the most sensitive full-length mRNA-seq data on the market, enabling the detection of low-expressed biomarkers that are missed by current 3′ end-counting technologies.
The Shasta system will be demonstrated at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting from April 5–10, 2024, where two posters have been accepted. Attendees of the AACR (Free AACR Whitepaper) meeting can learn more about Shasta workflows and view the system at Booth #2955. The Shasta system will also be presented at the Association of Biomolecular Resource Facilities (ABRF) Annual Meeting from April 21–24, 2024. At ABRF, attendees can engage with Takara Bio during the plenary keynote session on April 21st and again during the technology showcase at 8 am on April 23rd. Attendees can view the Shasta system at Booth #502. Researchers and other professionals wanting details can sign up to receive priority access to product and technical information.

On April 2, 2024 Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) reported that two poster presentations highlighting ongoing global clinical studies in its oncology pipeline will be presented at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024 taking place April 5-10, 2024, in San Diego, California (Press release, Zai Laboratory, APR 2, 2024, View Source [SID1234641727]).

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The global oncology programs to be showcased at AACR (Free AACR Whitepaper) 2024 include a Phase 1a/1b study of ZL-1310 (NCT06179069), a novel antibody-drug conjugate (ADC) within the Zai Lab pipeline that targets the Delta-like ligand 3 (DLL3), a validated therapeutic target for the treatment of small cell lung cancer (SCLC). ZL-1310 is designed with a novel linker-payload platform TMALIN which leverages the tumor microenvironment to overcome challenges associated with first-generation ADC therapies.

Also featured at AACR (Free AACR Whitepaper) 2024 will be Zai Lab’s Phase 1 study (NCT05859464) of ZL-1218, an anti-CCR8 antibody that blocks regulatory T cells (Treg) which suppress antitumor immunity in tumor tissue and is designed to deplete Treg cells selectively in tumors and minimally in other tissue.

"These ongoing global clinical studies underscore Zai Lab’s continued commitment to pursue both novel and validated cancer biology targets and advance innovative oncology therapies that can potentially reach patients around the world," said Rafael G. Amado, M.D., President, Head of Global Oncology Research and Development, Zai Lab. "We are dedicated to advancing the pipeline through both drug discovery and partnerships to address therapeutic challenges and unmet patient needs. As we extend the impact of our R&D innovation on a global scale, we look forward to highlighting these programs from our differentiated oncology pipeline at AACR (Free AACR Whitepaper) 2024."

Details regarding the Zai Lab poster presentations at AACR (Free AACR Whitepaper) 2024 are as follows:

Title: Trial in Progress: A Phase 1a/1b, An Open-label, Multicenter Study of ZL-1310 to Evaluate the Safety, Tolerability, and Pharmacokinetics in Subjects with Small Cell Lung Cancer
Presenter: Linda Liu, Ph.D., Senior Vice President, Biologics Discovery, Zai Lab
Session: PO.CTP01.02 – Phase I Clinical Trials in Progress 2, CT155 / 6
Date/Time: Monday, April 8, 2024, 1:30 PM – 5:00 PM PT
Location: San Diego Convention Center

Title: Trial in Progress: A Phase I, Open-label, Multicenter Study of ZL-1218, an anti-CCR8 IgG1, as a Single Agent and as Combination Therapy with Anti-PD-1 Antibody to Evaluate the Safety, Tolerability, and Pharmacokinetics in Subjects with Advanced Solid Tumor
Presenter: Maria Tea, M.D., Senior Medical Director, Zai Lab
Session: PO.CTP01.02 – Phase I Clinical Trials in Progress 2, CT162 / 13
Date/Time: Monday, April 8, 2024, 1:30 PM – 5:00 PM PT
Location: San Diego Convention Center

On April 2, 2024 TME Pharma N.V. (Euronext Growth Paris: ALTME), a biotechnology company focused on developing novel therapies for treatment of cancer by targeting the tumor microenvironment (TME), reported that the US Food and Drug Administration (FDA) has granted Fast Track designation for NOX-A12 (olaptesed pegol), TME Pharma’s CXCL12 inhibitor, in combination with radiotherapy and bevacizumab for use in the treatment of the aggressive adult brain cancer, glioblastoma, in the newly diagnosed setting where the tumor is resistant to chemotherapy and measurable tumor remains after surgery (Press release, TME Pharma, APR 2, 2024, View Source [SID1234641726]).

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The FDA’s Fast Track designation aims to bring important new drugs to patients more quickly, facilitating the development and expediting the review of therapies intended to treat serious conditions and address unmet medical needs. Companies whose programs are granted Fast Track designation can benefit from more frequent interactions with the FDA during the clinical development process and potentially "accelerated approval" and "priority review" if the relevant criteria are met.

TME Pharma continuously evaluates ways to advance the clinical development of NOX-A12 while remaining focused on identifying and securing financial resources from multiple sources, including those having no or minimal dilutive effect on its shareholders, such as governmental grants or free supply of combination drugs. In addition to engaging with industry partners and specialized healthcare investors, TME Pharma will also explore the eligibility of NOX-A12-based therapy for compassionate use programs once sufficient Phase 2 data have been generated. The company would prioritize such programs that support financial compensation for therapies leading to revenue generation, thus potentially reducing the financial needs of late-stage clinical development and also helping to generate real-world clinical evidence.

Recently announced clearance by the FDA of TME Pharma’s Investigational New Drug (IND) application for a Phase 2 study with NOX-A12 in glioblastoma, that the company plans to initiate later this year, was a prerequisite to having Fast Track designation granted by the FDA. Having Fast Track designation in addition to an open IND with an FDA-approved study design that addresses questions of dosing and contribution of components optimizes late phase development and offers an economically efficient model which further de-risks TME Pharma’s glioblastoma program. Following IND approval, this Fast Track designation is an external validation of NOX-A12’s potential to address the unmet need for glioblastoma patients.

The necessary preparatory steps for the NOX-A12 Phase 2 in glioblastoma are ongoing, and TME Pharma is aiming to initiate the new Phase 2 study as soon as the necessary resources from financial and industrial partners have been secured. TME Pharma is prioritizing discussions with partners willing to support the company over the long term and having their financial interests aligned with current stakeholders. While discussions are ongoing, and until longer-term agreements with partners are reached, TME Pharma is determined to keep operational costs low to extend the financial visibility as far as possible and increase the chance of success.

TME Pharma’s latest regulatory milestones were supported by recent survival data from the GLORIA Phase 1/2 study in which NOX-A12 demonstrated an unprecedented median Overall Survival of 19.9 months in chemotherapy resistant patients with residual tumor after surgery, which compared very favorably to a matched standard of care reference cohort and exceeds what TME Pharma believes to be all relevant competitor therapy trials in newly diagnosed glioblastoma patients resistant to standard chemotherapy.

"At the start of this year, we announced the next phase of our development of NOX-A12 by targeting IND approval and an expedited regulatory pathway in the US and we are very proud to have successfully achieved these milestones within the timeframe we set out," said Aram Mangasarian, CEO of TME Pharma. "While advancing discussions with potential industrial and financial partners may require some time to materialize, the open IND and Fast Track designation awarded by the FDA are well-received signals by these partners. We now have a clear clinical development roadmap with which to take NOX-A12 forward in the treatment of glioblastoma and to support engagement with potential partners. We expect our new Phase 2 study will build on the unprecedented results of our GLORIA trial, which strengthens the potential of NOX-A12 to become the treatment option of choice for newly diagnosed chemotherapy-resistant glioblastoma. We look forward to working closely with the FDA as we advance NOX-A12 to market as quickly as possible for the benefit of patients suffering from this devastating and aggressive cancer for which there is extremely poor prognosis."

On April 2, 2024 Eureka Therapeutics, Inc., a clinical-stage biotechnology company developing novel T-cell therapies to treat cancer, reported the activation of UCSF Benioff Children’s Hospitals as the first California site to join the ARYA-2 clinical trial (NCT04634357) (Press release, Eureka Therapeutics, APR 2, 2024, View Source [SID1234641725]).

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The ARYA-2 trial is evaluating Eureka’s investigational ARTEMIS ET140203 T-cell therapy which targets the alpha-fetoprotein (AFP)-peptide/HLA-A2 complex. This therapy is designed to treat pediatric subjects (1 to 21 years) with relapsed or refractory liver cancer, including hepatoblastoma (HB), hepatocellular neoplasm not otherwise specified (HCN-NOS), and hepatocellular carcinoma (HCC).

"Multiply relapsed or refractory liver cancers in children and adolescents are highly complex and lack effective treatment options, especially given the rarity of these conditions," said Dr. Arun Rangaswami, MD, Professor of Clinical Pediatrics and a senior solid tumor faculty member at UCSF. "We are excited to collaborate with Eureka to bring the promise of next-generation engineered T-cell therapy to this pediatric population with high unmet medical needs."

"We are proud to contribute to the collaborative effort aimed at developing cutting edge therapies for treating pediatric cancer," said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development, at the California Institute for Regenerative Medicine (CIRM). "Our recent $10.6 million grant towards Eureka’s ARYA-2 program underscores our commitment to improving the lives of young patients battling cancer."

"Our ARTEMIS T-cells are designed to overcome challenges in safely and effectively treating solid tumors," said Dr. Cheng Liu, President and CEO of Eureka Therapeutics. "UCSF’s participation significantly accelerates progress in bringing this potentially life-saving therapy to young patients with liver cancer."

This expansion builds upon Eureka’s ongoing efforts with the ARYA-2 trial, which is also actively recruiting patients at the Dana-Farber Cancer Institute. For more information on the ARYA-2 trial, Eureka’s T-cell therapy platform, and the potential of this treatment, please visit eurekaconnectme.com.

On April 2, 2024 Kelonia Therapeutics, a biotech company revolutionizing in vivo gene delivery, reported new preclinical data from its lead program KLN-1010, which will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024 in San Diego, California (Press release, Kelonia Therapeutics, APR 2, 2024, View Source [SID1234641724]). KLN-1010, a novel, in vivo CAR-T cell therapy candidate for the treatment of multiple myeloma, which leverages the company’s in vivo Gene Placement System (iGPS) technology, demonstrated that it is safe and effective in preclinical animal models.

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"These data reinforce the best-in-class potential of our in vivo CAR-T cell therapies," said Kevin Friedman, Ph.D., Chief Executive Officer and Founder of Kelonia. "By eliminating the need for ex vivo manufacturing and toxic lymphodepleting chemotherapy, we believe our iGPS technology will remove barriers that currently prevent patients from accessing transformative genetic medicines. We are excited by the preclinical profile of KLN-1010, and are looking forward to advancing this program towards the clinic in the near future."

Diverse T cell lineages, including memory and effector CD4 and CD8 T cells were modified in vivo by KLN-1010 to express a fully human anti-BCMA CAR, without detectable transduction of multiple myeloma tumor cells. A single intravenous injection of KLN-1010 displayed potent anti-tumor efficacy and caused complete tumor regression at multiple dose levels in several preclinical animal models. In other studies, treatment of non-human primates with an iGPS particle expressing an anti-CD20 CAR resulted in potent CAR-T cell activity lasting several months without the need for additional treatments or conditioning chemotherapy.

Details for the poster presentation are as follows:

Title: T cell-specific in vivo transduction with preclinical candidate KLN-1010 generates BCMA directed CAR T cells with potent anti-multiple myeloma activity
Poster Session: Adoptive Cell Therapies 2: CAR-T Cells
Date and Time: April 7, 2024 at 1:30-5:00 pm PT
Location: Section 2
Poster Number: 16