On September 26, 2024 Laverock Therapeutics, a gene-silencing company with a uniquely powerful technology for the development of programmable advanced therapies to tackle major diseases, reported significant progress in the industrialisation and validation of its technology platform enabling broadening of its potential therapeutic application. Laverock also confirms in house focus on the development of a pipeline of programmable advanced therapies in oncology and genetic disease including Charcot-Marie-Tooth disease.

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In the course of the last year, Laverock has generated a complete set of data in human cells demonstrating the unique features of its technology – namely the ability to silence genes in a programmable, tunable, stable and highly specific manner across a number of target genes and cell types. Laverock’s initial data showcasing the unique properties of the technology was generated against a single target (B2M, a component of the MHC-I complex), and the Company has now designed and validated silencing RNAs against seven additional targets, demonstrating that the technology is widely applicable and not limited to specific targets or cell types.

In addition, Laverock has demonstrated that the technology can be used to concomitantlysilence more than one target gene, further expanding the range of its potential therapeutic applications. Further data now also supports the use of our gene-silencing approach to control the delivery of payloads and thereby use cells to deliver biologics in a programmed manner.

To drive our in-house product development and partnering, Laverock has also materially increased the robustness and throughput of its platform with significant increases in capacity to screen and validate constructs and carry out gene editing and cell line development.

The company will be presenting these data at a number of conferences in the coming months.

In the light of the growing body of evidence supporting the Laverock platform’s unique attributes, and the platform’s capacity to rapidly advance programmes, Laverock has solidified its in house therapeutic focus on high value areas, adding genetic diseases to oncology.

To maximise the value of its platform Laverock is actively seeking partners for indications where it has already generated product concepts, for example Type 1 diabetes/conditional hypoimmunogencity, and for new programmes in other indications including regenerative medicine, fibrosis and inflammation.

David Venables, CEO of Laverock, said: "Over the course of this year, following our seed financing last year, we have made enormous progress in validating our platform, proving its versatility and wide applicability. We have also ensured that we have a robust, industrialised process capable of rapidly advancing product concepts. We believe we are uniquely positioned to generate powerful and entirely novel advanced therapies across a range of disease areas through advancing our own oncology programmes to the clinic as rapidly as possible, generating pipeline depth with further programmes in genetic diseases, and through strategic partnering."

Laverock has commenced a financing round to fund its pipeline development and further advance the platform.

(Press release, Laverock Therapeutics, SEP 26, 2024, View Source [SID1234668717])

On September 26, 2024 OSE Immunotherapeutics reported its consolidated half-year financial results and provided updates on key milestones achieved during the H1 2024 as well as the Company’s outlook for its immunotherapies in immuno-oncology and immuno-inflammation (Press release, OSE Immunotherapeutics, SEP 26, 2024, View Source [SID1234646993]).

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Nicolas Poirier, Chief Executive Officer of OSE Immunotherapeutics, comments: "The major milestones achieved during H1 2024 are paving the way for a transformative year for OSE. During this period, thanks to the OSE teams, the Company made significant outstanding progress.

The half-year has seen continued execution of our partnership-focused business model through three strategic pharmaceutical agreements with major partners, AbbVie and Boehringer Ingelheim, related to our differentiated immunological pipeline. These key achievements trigger a solid financial position supporting the Company’s growth, relying on our promising clinical and preclinical proprietary programs in immuno-inflammation and immuno-oncology conducted and supported by highly skilled OSE teams

We also achieved two significant inflection points on our late-stage proprietary clinical assets. In immunoinflammation, the positive clinical efficacy and safety results for Lusvertikimab in ulcerative colitis represent a strong catalyst for potential future partnership opportunities. We have generated exciting data that we plan to communicate with our investigators at an upcoming global medical conference. In immuno-oncology, the international registration study Artemia for cancer vaccine Tedopi in second-line non-small cell lung cancer treatment is now on track globally. In parallel, in order to ensure continuous portfolio development, we continue accelerating and strengthening first-in-class preclinical programs from our innovative research platforms".

Anne-Laure Autret-Cornet, Chief Financial Officer of OSE Immunotherapeutics, said: "With more than €90 million non-dilutive cash-in in 2024, our financial visibility is strongly reinforced until 2027. This allows us to prioritize funding of our recently globally launched Artemia Phase 3 registration study for our cancer vaccine Tedopi in lung cancer and to further invest in our other proprietary clinical products and innovative R&D engine to increase the value and interest of our assets."

On September 26, 2024 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of therapeutic biologics to selectively engage and modulate disease-specific T cells, reported the pricing of an underwritten public offering of (i) 11,564,401 shares of its common stock and accompanying common stock warrants to purchase an aggregate of 2,891,100 shares of common stock and, (ii) to certain investors in lieu of common stock, pre-funded warrants to purchase 12,435,599 shares of common stock and accompanying common stock warrants to purchase an aggregate of 3,108,900 shares of common stock (Press release, Cue Biopharma, SEP 26, 2024, View Source [SID1234646893]). Each share of common stock and accompanying common stock warrant are being sold together at a combined public offering price of $0.50, and each pre-funded warrant and accompanying common stock warrant are being sold together at a combined public offering price of $0.499. The aggregate gross proceeds of the offering are expected to be approximately $12.0 million, before deducting underwriting discounts and commissions and other offering expenses. Each pre-funded warrant will have an exercise price of $0.001 per share, will be exercisable immediately and will be exercisable until all of the pre-funded warrants are exercised in full. Each common stock warrant will have an exercise price of $0.50 per share, will be exercisable immediately and will expire five years from the date of issuance. The offering is expected to close on or about September 30, 2024, subject to satisfaction of customary closing conditions. All of the securities are being offered by Cue Biopharma.

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Oppenheimer & Co. Inc. is acting as sole book-running manager for the offering. Newbridge Securities Corporation is acting as co-manager for the offering.

A shelf registration statement on Form S-3 (File No. 333-271786) relating to the securities to be offered in the public offering was filed with the Securities and Exchange Commission (the "SEC") on May 9, 2023 and declared effective on May 26, 2023. The offering was made only by means of a prospectus supplement and accompanying prospectus that form a part of the registration statement. A preliminary prospectus supplement relating to and describing the terms of the offering has been filed with the SEC and may be obtained for free by visiting the SEC’s website at www.sec.gov. A final prospectus supplement relating to the offering will be filed with the SEC. When available, copies of the preliminary prospectus supplement and final prospectus supplement relating to the offering may also be obtained by contacting: Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, New York 10004, by telephone at (212) 667-8055, or by email at [email protected].

This press release does not constitute an offer to sell, or a solicitation of an offer to buy these securities, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On September 26, 2024 Mission Bio, a leader in single-cell multiomics solutions for precision medicine, reported the launch of its Tapestri Genome Integrity CNV Solution (Press release, Mission Bio, SEP 26, 2024, View Source [SID1234646890]). This new product is the only single-cell high-throughput solution for measuring genome-wide copy number variants (CNVs) on the market and is designed to fulfill important needs in the critical areas of therapeutic development oncology research.

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While genome-editing and stem cell therapies continue to harbor tremendous promise for treating complex diseases but have encountered safety concerns associated with genomic instability. The latest FDA guidance suggests that assessment of genomic integrity should include chromosomal abnormalities like CNVs (i.e., duplications and deletions) as indicators of clinical safety. Conventional methods of assessing genomic instability such as g-banding fail to provide adequate answers due to their low throughput, as they may not analyze enough cells to detect aberrations. Oncology researchers have faced similar issues unlocking the potential of CNVs to serve as prognostic or therapeutic markers associated with tumor evolution, immune evasion, and therapeutic resistance. Existing bulk techniques provide only a limited view of CNV-based clonal architecture.

Mission Bio’s Tapestri Genome Integrity CNV Solution enables both CGT developers and oncology researchers with high-throughput, single-cell multiomic analysis of genome-wide CNVs, combined with automated reporting and multiplexing capabilities to ensure even more accessibility. Compared to current methods, the solution equips CGT developers with improved assay throughput to uncover potentially adverse chromosomal events, while simultaneously measuring genome editing outcomes. For oncology researchers, the solution enables the assessment of cell-to-cell aneuploidy and CNV events across the genome with the potential to co-measure SNVs and focal CNVs. Tapestri enables these researchers the rapid characterization of clonal heterogeneity behind tumorigenesis and therapy resistance.

"While working on a new way of investigating chromosomal instability and aneuploidy within tumors, we found that Mission Bio’s Tapestri Platform was able to provide the combination of high throughput and high sensitivity we needed," said Dr. Teresa Davoli of the NYU School of Medicine’s Institute for Systems Genetics and Department of Biochemistry and Molecular Pharmacology. "Mission Bio’s Tapestri platform gave our team the flexibility and customizability necessary to push our understanding of aneuploidy across thousands of single cells simultaneously." For details of her study see "KaryoTap Enables Aneuploidy Detection in Thousands of Single Human Cells."

"The launch of the Tapestri Genome Integrity CNV Solution represents a significant advancement in therapeutic development and cancer research," said Vanee Pho-Conners PhD, Senior Director of Product Management at Mission Bio. "By offering genome-wide CNV analysis at the single-cell level, we’re bridging a critical safety gap for CGT developers, who require precise genomic stability measurements to ensure the safety and efficacy of their therapies. This innovation highlights our commitment to equipping researchers with the most comprehensive tools to drive transformative breakthroughs in treatment."

The assay will be a key focus of Mission Bio at the 31st annual European Society of Gene & Cell Therapy Meeting, taking place Oct. 22-25 in Rome. For more information, please visit booth B24 or View Source

On September 26, 2024 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine and patient care, reported an expansion to its collaboration with Takeda (TSE:4502/NYSE:TAK) that takes a data-first approach to research and development, with the aim of enhancing Takeda’s oncology research and development efforts (Press release, Tempus, SEP 26, 2024, View Source [SID1234646889]).

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After an initial collaboration that leveraged Tempus’ de-identified multimodal datasets, this new agreement will expand upon that work. Takeda will combine Tempus’ multimodal real-world datasets and Tempus’ biological modeling capabilities to advance Takeda’s pipeline of cancer therapeutics, which includes antibody-drug conjugates (ADCs), small molecules, bispecifics and gamma delta T-cell therapies.

Takeda researchers will use Tempus’ analytics platform, Lens, to gain real-time access to de-identified patient records as well as a suite of AI-enabled tools to accelerate critical insights for Takeda’s drug development efforts. Initial work has resulted in research that will be presented at an upcoming medical congress.

In parallel, the two companies have embarked on a multi-phase biological modeling project leveraging one of the largest repositories of patient-derived tumor organoids in the world. Each organoid model can be used to reflect the biology of a distinct patient tumor, and then be genetically linked through Tempus’ next-generation sequencing assays to Tempus’ real-world multimodal dataset, potentially enhancing the predictability of a drug’s effectiveness. This approach enables efficient hypothesis generation and rapid validation that could guide early drug candidate prioritization. For this unique scope of work, Tempus is working with Takeda on a panel of 60 organoids spanning 10 cancer indications that closely reflect real-world patients to functionally evaluate several preclinical candidates.

"We are excited to expand our relationship with Takeda, combining real-world multimodal data and biological modeling capabilities to better understand targets of interest," said Ryan Fukushima, Chief Operating Officer at Tempus. "The results we’ve seen thus far demonstrate how the collaboration between the Takeda and Tempus teams can assist in efforts to accelerate Takeda’s growing oncology therapeutic pipeline, which may lead to the next generation of cancer treatments."