On January 13, 2025 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that it has received 510(k) clearance from the United States Food and Drug Administration (FDA) for its highly-sensitive in-situ hybridisation (ISH) test, the VENTANA Kappa and Lambda Dual ISH mRNA Probe Cocktail (Press release, Hoffmann-La Roche, JAN 13, 2025, View Source [SID1234649687]). The test is designed to help pathologists differentiate a B-cell malignancy from a normal, reactive response to an infection, thus facilitating faster access to treatment.4 This announcement follows the assay’s CE Mark approval in June 2024.

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B-cell lymphoma is a type of cancer that typically develops in the lymphatic system. It accounts for approximately 85 percent of non-Hodgkin lymphoma (NHL) cases.3 NHL is one of the most common forms of cancer in the US, accounting for about 4% of all cancer cases,5 and causing more than 80,000 deaths each year.5 In the early stages of NHL, patients may experience symptoms like swelling of the lymph nodes, fever, fatigue, loss of appetite or a red rash.

"Accurately differentiating lymphoma from an infection is critical in ensuring accurate and timely diagnosis, especially as the symptoms can appear similar," said Jill German, Head of Pathology Lab at Roche Diagnostics. "With this new test, clinicians can have confidence in their diagnosis, while the test reduces the need for multiple samples and time consuming follow up tests, giving patients certainty sooner, and enabling faster access to the right treatment."

With increased sensitivity, the new VENTANA Kappa and Lambda Dual ISH mRNA Probe Cocktail enables assessment across the more than 60 B-cell lymphoma subtypes and plasma cell neoplasms on a single tissue slide. The test can assess small biopsies and formalin-fixed tissue, reducing the need for a fresh tissue sample, which may not be available especially if lymphoma was not originally suspected. These test properties preserve tissue, may result in fewer additional patient biopsies and make interpretation quicker and easier for the pathologist, helping facilitate a faster diagnosis and access to treatment for patients.

This first-of-its-kind assay is a significant addition to Roche’s industry-leading hematopathology portfolio, which includes more than 65 biomarkers.

About the VENTANA Kappa and Lambda Dual ISH mRNA Probe Cocktail
The VENTANA Kappa and Lambda Dual ISH mRNA Probe Cocktail Assay is a qualitative assay that is used to detect the expression of kappa and lambda immunoglobulin light chains in formalin-fixed paraffin embedded (FFPE) human hematolymphoid specimens by in situ hybridization (ISH).

The assay is intended as an aid in the diagnosis of mature B-cell lymphomas and plasma cell neoplasms. The VENTANA Kappa and Lambda Dual ISH mRNA Probe Cocktail is indicated for use when a biopsy of lymph node or bone marrow (core biopsy and clot section) indicates inconclusive results. It enables the assessment of both markers in the context of one another on a single slide as an aid in differentiating between a reactive process or B-cell lymphoma and plasma cell neoplasms.

This is not a standalone test, and results should be evaluated by a qualified pathologist within the context of the patient’s clinical history and other diagnostic tests. This product is intended for in vitro diagnostic (IVD) use.

On January 13, 2025 Ginkgo Bioworks (NYSE: DNA), which is building the leading platform for cell programming and biosecurity, reported a research collaboration with Universal Cells, an Astellas company, to optimize next-generation induced pluripotent stem cell (iPSC)-derived cancer cell therapies (Press release, Ginkgo Bioworks, JAN 13, 2025, View Source [SID1234649686]). This collaboration underscores Ginkgo’s capacity to deploy high-throughput biological approaches to tackle the complex challenge set associated with development of therapies targeting solid tumors.

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iPSC-derived cell therapies have the potential to transform cancer care by offering scalable, ‘off-the-shelf’ treatment options. However, bringing efficacious therapies to patients requires robust design and screening processes to improve persistence in the patient to enable durable clinical responses. By combining Universal Cells’ proprietary iPSC-derived cell technologies with Ginkgo’s expertise in design and screening large CAR libraries and its high throughput, multimodal immune cell engineering platform, the companies aim to accelerate the development of more potent and durable cell therapies while maintaining manufacturability at scale.

Ginkgo’s approach leverages computational tools, deep expertise in library assembly and screening, and rigorous statistical analysis and biological insight to help partners discover and engineer optimal therapeutic designs with greater speed and precision. Ginkgo’s platform capabilities extend across multiple dimensions of cell therapy development, including chimeric antigen receptor (CAR) discovery and optimization, armoring strategies for enhanced cell survival and persistence, and gene editing tools for immune cell engineering. Ginkgo employs high-throughput pooled and arrayed screening methods that enable the simultaneous testing of numerous CAR designs from transcriptome to cell function. This approach facilitates discoveries that aim to improve immune cell persistence and functionality.

"We’re excited to embark on this collaboration, which represents a significant milestone for Ginkgo as we expand our cell therapy portfolio," said Jason Kelly, CEO and co-founder of Ginkgo Bioworks. "Teaming up with Universal Cells, part of Astellas, allows us to bring our comprehensive immune cell engineering capabilities to a highly respected, global pharmaceutical leader. We look forward to working together to optimize iPSC-derived allogeneic cell therapies that have the potential to improve patient outcomes and reshape how we treat solid tumors."

"Cell therapies hold immense promise, but realizing their full potential requires innovation at every step, from design to delivery," said Dr. Narendra Maheshri, VP, Genetic Medicines at Ginkgo Bioworks. "Our platform enables partners to rapidly explore the functional impact of a large number of CAR designs simultaneously, and then iterate to further refine those designs. By applying this approach with Universal Cells, we aim to help accelerate CAR approaches, opening up new possibilities in the fight against cancer."

To learn more about how you can leverage Ginkgo’s capabilities in cell therapy and gene editing – from CAR-T to CRISPR and beyond – please visit our website.

On January 13, 2025 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, reported that KJ-C2219, an allogeneic CAR T-cell therapy targeting CD19/CD20, has administered the first dose to a patient in an investigator-initiated trial (IIT) (Press release, Carsgen Therapeutics, JAN 13, 2025, View Source [SID1234649685]).

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KJ-C2219 is developed based on CARsgen’s THANK-u Plus platform and is designed for the treatment of hematologic malignancies and autoimmune diseases. An investigator-initiated trial is ongoing in China to evaluate KJ-C2219 for the treatment of relapsed/refractory B-cell non-Hodgkin lymphoma (R/R B-NHL).

About THANK-u Plus

CARsgen has developed the THANK-u Plus platform as an enhanced version of its proprietary THANK-uCAR allogeneic CAR-T technology to address the potential impact of NKG2A expression levels on therapeutic efficacy. THANK-u Plus demonstrates sustained expansion regardless of varying NKG2A expression levels on NK cells and exhibits significantly improved expansion compared to THANK-uCAR. Preclinical studies show that THANK-u Plus delivers superior antitumor efficacy in the presence of NK cells compared to THANK-uCAR. Allogeneic BCMA or dual-targeting CD19/CD20 CAR-T cells developed using this platform exhibit robust antitumor activity in the presence of NK cells, indicating that THANK-u Plus has broad potential for developing diverse allogeneic CAR-T therapies.

On January 13, 2025 SciTech Development, a clinical-stage pharmaceutical company specializing in oncology, reported the initiation of the next stage of its clinical trial for their lead drug candidate, ST-001 nanoFenretinide (ST-001) (Press release, SciTech Development, JAN 13, 2025, View Source [SID1234649684]). This milestone follows the successful completion of the accelerated safety and dose-escalation stage, where ST-001 demonstrated exceptional safety, tolerability, and pharmacokinetic performance.

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Advancing the Phase 1a Trial with Clear Clinical Objectives

The continuation of the ST-001 clinical trial aims to achieve several critical clinical objectives:

Confirm that ST-001 achieves previously reported clinical response rates.
Determine the pharmacological profile of intravenous fenretinide delivered using SciTech’s novel nanoparticle delivery system, designed for drugs with poor water solubility.
Determine the recommended treatment dose (RTD) to evaluate disease activity and response rates in the follow-on Phase 1b trial and the planned trial in Small Cell Lung Cancer.
"The success of our accelerated Phase 1a trial marks a pivotal milestone, driving ST-001 into the next critical stage of development," said Earle Holsapple, CEO of SciTech Development. "We are thrilled about the potential of ST-001 nanoFenretinide and its promise to transform treatment outcomes for patients battling cancers such as T-cell Non-Hodgkin Lymphoma."

About ST-001 nanoFenretinide

ST-001 nanoFenretinide is a patented nanoparticle IV formulation that combines the drug fenretinide with biocompatible phospholipids. This groundbreaking new investigational drug overcomes bioavailability challenges, enhances therapeutic efficacy, and minimizes toxicity in cancer patients. Powered by SciTech’s advanced delivery platform, ST-001 enables rapid delivery of effective doses of fenretinide to cancer cells, marking a breakthrough in hard-to-treat cancers.

Encouraging Early Results in Phase 1a Accelerated Trial

During the accelerated stage of the trial, ST-001 achieved significant milestones:

Patient Enrollment is Complete for the accelerated stage of the clinical trial marking a significant accomplishment for patients with this rare disease.
Therapeutic Responses Observed: Patients demonstrated partial responses and stable disease, marking an important step for trial advancement.
Excellent Tolerability for ST-001 across all dose levels to date.
Outstanding Pharmacokinetics: The nanoparticle delivery platform overcame longstanding bioavailability challenges, ensuring efficient delivery of fenretinide to tissues.
Future Direction for ST-001 nanoFenretinide

Building on the success of the accelerated Phase 1a stage, the trial begins detailed evaluation of clinical activity and response rates in T-cell lymphomas. The trial is actively underway at nine renowned medical centers across the country. Click here to learn more about the locations and patient trial criteria. Additionally, a separate trial targeting Small Cell Lung Cancer utilizing ST-001 is scheduled to begin in Q2 2025.

On January 13, 2025 Rappta Therapeutics ("Rappta"), focused on developing first-in-class anti-cancer drugs activating protein phosphatase 2A (PP2A), reported an exclusive global license agreement with SpringWorks Therapeutics ("SpringWorks") for RPT04402, a first-in-class molecular glue of specific Protein Phosphatase 2A (PP2A) complexes (Press release, Rappta Therapeutics, JAN 13, 2025, View Source [SID1234649683]).

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Rappta’s PP2A-reactivating technologies developed using its proprietary high resolution structural data have the potential to create a new class of anti-cancer drugs for treating a broad range of human cancers. PP2A is a critical enzyme in regulating protein de-phosphorylation and its reactivation is fundamental for tumor suppression, but it has been historically hard to target.

PP2A mutations are oncogenic drivers in molecularly defined subsets of uterine cancer and represent a targetable subset of patients with a high unmet clinical need. In pre-clinical models of PP2A mutant uterine cancer, RPT0402 achieved rapid, deep and durable tumor regressions at as monotherapy.

Under the exclusive license agreement, SpringWorks Therapeutics will be responsible for global development and commercialization of RPT04402. SpringWorks has paid Rappta $13 million upfront, and Rappta is also eligible to receive further clinical, regulatory and commercial milestone payments, and tiered single-digit royalties on net sales. SpringWorks expects to file an Investigational New Drug (IND) application for RPT04402 by the end of 2025.

Sunjeet Sawhney, Chief Executive Officer of Rappta Therapeutics, commented: "Rappta is the only company to have successfully targeted PP2A, a notoriously difficult and undruggable target. Data we generated demonstrated the potential of RPT04402 for treating large, underserved patient populations. SpringWorks, a leader in the targeted oncology space, has the expertise and knowledge to accelerate the further development of our first in class asset. I would like to thank our team and investors who have supported our journey and we look forward to following the progress made in this area"

Goutham Narla, Chief Scientific Officer of Rappta Therapeutics and Professor of Internal Medicine and Human Genetics at the University of Michigan, said: "Our team at Rappta Therapeutics has been able to leverage our proprietary structural data to develop a first-in-class series of molecular glues to the previously undruggable tumor suppressor PP2A. We are excited to be able to work with SpringWorks to potentially translate this approach to the clinic with the hope that this approach will have broad applications for the treatment of human cancers."

Rappta was founded by Goutham Narla & Mikko Mannerkoski, back in 2019 with funding from Novartis Venture Fund ("NVF"), Novo Holdings, Advent Life Sciences and a family office in Series A financing alongside other non-dilutive funding from Business Finland.