On May 18, 2023 Orna Therapeutics, a biotechnology company pioneering a new investigational class of engineered circular RNA (oRNA) therapies, presented data on the progress of lead program ORN-101, a development-stage in situ CAR program, at the Protein Engineering Summit (PEGS) and at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting in Los Angeles (Press release, OrnaTherapeutics, MAY 18, 2023, View Source [SID1234631852]).

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The presentations include a session and talk at PEGS, as well as a poster presentation at ASGCT (Free ASGCT Whitepaper), which may be viewed on the Orna website here.

"Using the Orna FoRCE platform, we’ve been able to develop ORN-101, a potent anti-cancer circular RNA therapy delivered via LNP," said Robert Mabry, PhD, Chief Scientific Officer at Orna. "We have achieved 20-fold lower dosing in animal models compared to earlier versions of the product, allowing us the flexibility to administer multiple doses of our off-the-shelf immunotherapy – something that could allow us to treat more patients quickly and easily."

Synthetic Circular RNA as a New Therapeutic Modality

Elevated and durable protein expression – two features where mRNA falls short – are maximized with circular RNA in part due to Orna’s discovery of novel internal ribosome entry site (IRES) elements necessary for translation. Compared to previously known IRES elements, Orna’s library of IRES elements can be used to drive higher protein expression in desired cell type targets. oRNA, because of its shape, is more resistant to quick degradation in the body, is easier to manufacture and formulate in the lab, and is immunoquiescent. These desirable features further allow oRNA therapeutics to be developed for applications beyond infectious disease, including oncology and genetic disorders.

In situ CAR Therapy Using oRNA

Over the past four years, Orna has worked to develop a therapeutic class capable of delivering a chimeric antigen receptor (CAR) to immune cells within a patient, eliminating the need for lymphodepletion typically required for engineered cell therapies, while providing off-the-shelf redosability in an autologous setting. ORN-101 combines oRNA and a proprietary LNP, and these latest data show ORN-101’s tumor eradication ability after 2-3 doses in a mouse model, effective at 10-20-fold lower doses than Orna has previously reported. ORN-101 features high expression of the CAR driven by an optimized IRES element, selected by Orna’s FoRCE platform to yield durable protein expression.

About ORN-101:

ORN-101, Orna’s lead program, is a development-stage in situ CAR therapy designed to modify a patient’s immune cells inside their body. Comprising an oRNA molecule packaged inside a proprietary lipid nanoparticle (LNP) formulation, this easily redosable format could avoid patient lymphodepletion and allow for reliable dose control, overcoming barriers of existing autologous ex vivo CAR-T therapies without sacrificing efficacy. Preclinical data demonstrates tumor suppression and eradication in animal models, suggesting the possibility that oRNA-LNP based cancer therapies could disrupt traditional CAR-T cellular therapies.

On May 18, 2023 Biodexa Pharmaceuticals PLC (NASDAQ: BDRX) ("Biodexa" or the "Company"), a clinical stage biopharmaceutical company developing a pipeline of products aimed at primary and metastatic cancers of the brain is pleased to announce that it has signed non-binding letter of intent to potentially acquire Varian Biopharmaceuticals, Inc. ("VarianBio"), a private US precision oncology company developing novel therapeutics for the treatment of cancer (Press release, Midatech Pharma, MAY 18, 2023, View Source [SID1234631849]).

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Background to the proposed acquisition

In recent communications, the Company has referenced a shift in strategy from a drug delivery company to a therapeutics company. Currently, the Company’s only clinical asset is MTX110 which is being developed for three intractable rare and orphan brain cancers. The Company’s intention is to add to, and diversify, its development pipeline with a continued focus on rare and orphan products and/or oncology therapeutics and VarianBio represents the first of such opportunity.

On May 18, 2023 Micronoma reported that it is serving as the industry partner on a grant the NYU Grossman School of Medicine received from the National Cancer Institute to identify microbial and host genomic signatures that can be used with other methodologies to predict non-small cell lung cancer (NSCLC) in its early stages as well as the chance of post-surgical recurrence (Press release, Micronoma, MAY 18, 2023, https://www.micronoma.com/micronoma-teams-up-with-nyu-school-of-medicine-on-lung-cancer-research-grant-this-latest-collaboration-seeks-synergistic-methodologies-that-will-enable-the-earliest-best-predictions-of-lung-cancer/ [SID1234631848]).

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"Micronoma is thrilled to partner again with NYU researchers to advance the cause of using microbial biomarkers to diagnose cancer as early as possible as well as establish potential recurrence markers," said Micronoma CEO and co-founder Sandrine Miller-Montgomery. "Lung cancer remains the leading cancer killer and the development of non-invasive methods to identify it is key to improving patient outcomes."

"This work will build on our groundbreaking work as the first biotech company offering early cancer detection using OncobiotaLUNG, a microbiome-driven liquid biopsy platform," she added.

Micronoma and NYU Langone Health are currently collaborating to use blood samples to derive multi-omics, including circulating tumor microbial DNA associated with early lung cancer diagnosis, which the company should make commercially available under CLIA regulations later this year.

With this grant, NYU and Micronoma intend to continue identifying the leading microbial and host biomarkers that can be used to predict early-stage lung cancer diagnosis and prognosis and use cutting-edge bioinformatic mechanisms and potentially targeted molecular tools to pursue developing early-stage testing and prognosis.

"We are extremely excited to bring a novel use of microbial genomic signatures as a potential biomarker for early-stage lung cancer to the Early Detection Research Network (EDRN) multicenter consortium," said Leopoldo N. Segal, M.D., Associate Professor of Medicine. "This work has been made possible through the collaborative efforts between NYU Langone Health and Micronoma. This is a promising area of research that we expect to be important in our efforts to diagnose early-stage lung cancer and risk stratify them."

As previously disclosed in the Current Report on Form 8-K filed by Iovance Biotherapeutics, Inc., a Delaware corporation (the "Company") with the Securities and Exchange Commission (the "SEC") on January 23, 2023, the Company and its newly formed, wholly owned subsidiary, Iovance Biotherapeutics UK Ltd ("Purchaser") entered into an Option Agreement (the "Option Agreement") with Clinigen Holdings Limited, Clinigen Healthcare Limited, and Clinigen, Inc. (collectively, "Clinigen"), pursuant to which the Purchaser would acquire the worldwide rights in Proleukin (aldesleukin) (the "Product"), as well as the manufacturing, supply, commercialization and the generation of income from such rights and associated operations from Clinigen (the "Acquisition") (Filing, Iovance Biotherapeutics, MAY 18, 2023, View Source [SID1234631847]).

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On May 18, 2023, the Company completed the Acquisition and specifically acquired (i) all issued and outstanding shares of Clinigen SP Limited (the "Target"), (ii) the business of the Target and Clinigen comprising the manufacturing, supply, commercialization and the generation of income from the Product rights and the undertaking of an active role in the development, maintenance and exploitation of those rights, and (iii) certain specified assets identified in the Option Agreement. The closing contemplated by the Option Agreement followed the satisfaction or waiver of the closing conditions under the Option Agreement, including the required submission of the premerger notification and report form filing under the Hart-Scott-Rodino Antitrust Improvement Act of 1976 to the Regulatory Authority (as defined in the Option Agreement).

Pursuant to the Option Agreement, the Company paid to Clinigen (i) an upfront payment of £167.7 million (or approximately $200 million) and (ii) a payment for certain inventory of £2.4 million (or approximately $3.1 million) using existing cash on hand. The Option Agreement also provides for a £41.7 million (or approximately $50 million) milestone payment upon first approval of lifileucel in advanced melanoma, and deferred consideration based on double digit rates on global net sales (as defined therein) payable from the Company to Clinigen following the completion of the Acquisition for the applicable deferred consideration term.

The foregoing description of the Option Agreement does not purport to be complete and is qualified in its entirety by reference to the Option Agreement, a copy of which was filed as Exhibit 10.1 to Amendment No. 1 to the Current Report on Form 8-K filed by the Company with the SEC on January 27, 2023.

On May 18, 2023 Geron Corporation (Nasdaq: GERN) reported that it has granted non-statutory stock options to purchase an aggregate of 128,840 shares of Geron common stock as inducements to newly hired employees in connection with commencement of employment with the Company (Press release, Geron, MAY 18, 2023, View Source [SID1234631846]).

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The stock options were granted on May 17, 2023 at an exercise price of $3.02 per share, which is equal to the closing price of Geron common stock on the date of grant. Stock options representing an aggregate of 122,220 shares have a 10-year term and vest over four years, with 12.5% of the shares underlying the options vesting on the six-month anniversary of commencement of employment for the respective employees and the remaining shares vesting over the following 42 months in equal installments of whole shares, subject to continued employment with Geron through the applicable vesting dates. Stock options representing an aggregate of 6,620 shares have a 10-year term and vest in full upon achievement of certain regulatory milestones, subject to continued employment with Geron through the applicable vesting dates. All of the stock options were granted as material inducement to employment in accordance with Nasdaq Listing Rule 5635(c)(4) and are subject to the terms and conditions of the stock option agreements covering the grants and Geron’s 2018 Inducement Award Plan, which was adopted December 14, 2018 and provides for the granting of stock options to new employees.