On April 23, 2024 Factor Bioscience Inc., a Cambridge-based biotechnology company focused on developing mRNA and cell-engineering technologies, reported its participation in the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting to be held in Baltimore, MD from May 7-11, 2024 (Press release, Factor Bioscience, APR 23, 2024, View Source;cell-therapy-asgct-27th-annual-meeting-302124039.html [SID1234642256]). Factor will deliver seven presentations, representing the company’s most expansive presentation of data to date.

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"2024 marks the fifth year that Factor will present our latest data at the Annual Meeting of the ASGCT (Free ASGCT Whitepaper)," said Dr. Matt Angel, Co-Founder, Chairman and CEO of Factor. "This year promises to be particularly exciting, as we present our progress on deploying the powerful mRNA cell-engineering technologies that we have built at Factor over the last decade."

Dr. Kyle Garland, Director of Translational Science at Factor, commented, "We have been working hard over the last year to address several critical needs in our industry, including tissue-targeted delivery of nucleic acids, durable allogeneic cell therapies, and effective approaches for targeting solid tumors. We look forward to presenting our results in these and other areas next month at the ASGCT (Free ASGCT Whitepaper) Annual Meeting."

Details of the presentations are below:

"iMSCs Derived from mRNA-Engineered B2M-KO iPSCs Exhibit Enhanced Immunosuppressive Activity and Stealthing Features." -to be presented by Raven Hinkel on May 11 at 10:30 am, Novel Immune Effector Cell Manufacturing Oral Abstract Session, Ballroom 2.

"Sustained Transgene Expression in Engineered iPSC-Derived Tissue-Specific Cells." -to be presented by Taeyun Kim on May 10 from 12:00-7:00 pm, Targeted Gene Insertion Poster Session.

"Chromatin Opening Elements Mitigate Silencing of Transgenes During iPS Cell to Macrophage or Lymphocyte Differentiation." -to be presented by Claire Aibel on May 10 from 12:00-7:00 pm, Gene Targeting and Gene Correction New Technologies Poster Session.

"Targeted Insertion of HLA-E at the B2M Locus of mRNA-Reprogrammed iPSCs Facilitates the Development of Allogenic Cell Therapies with Enhanced Safety Features." -to be presented by Elizabeth Belcher on May 10 from 12:00-7:00 pm, Targeted Gene Insertion Poster Session.

"iPS-Derived Macrophages Engineered to Express IL-12 Enhance Cancer Cell Killing of a Multi-Cell Type Therapeutic Platform." -to be presented by Ian Hay on May 10 from 12:00-7:00 pm, Immune Targeting and Approaches with Genetically-Modified Cells and Cell Therapies Poster Session.

"A Library of Novel Polyvalent Ionizable Lipids for Targeted Co-Delivery of mRNA and DNA." -to be presented by Joey Pisano, Ph.D. on May 10 from 12:00-7:00 pm, Nonviral Therapeutic Gene Delivery and Synthetic/Molecular Conjugates Poster Session.

"Novel Polyvalent Ionizable Lipids Enable Targeted Delivery of mRNA to T Cells and Monocytes." -to be presented by Ariadna Lubinus on May 10 from 12:00-7:00 pm, Nonviral Therapeutic Gene Delivery and Synthetic/Molecular Conjugates Poster Session.
For more information about the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, visit annualmeeting.asgct.org.

On April 23, 2024 Pillar Biosciences, Inc., the leader in Decision Medicine, which develops and distributes next-generation sequencing (NGS) tests to localize testing and reduce time to treatment initiation and testing costs, reported that the U.S. Food and Drug Administration (FDA) has approved the company’s Premarket Approval (PMA) supplement application for its oncoReveal CDx pan-cancer solid tumor in vitro diagnostic (IVD) (Press release, Pillar Biosciences, APR 23, 2024, View Source [SID1234642255]). The approval expands the indication of oncoReveal Dx from EGFR & KRAS therapy selection in non-small cell lung cancer (NSCLC) and colorectal cancer (CRC) to now include general solid tumor profiling.

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"We are pleased to have expanded the clinical utility of oncoReveal Dx, which was the first FDA PMA-approved multi-cancer IVD kit launched in the market," said Gang Song, Founder and Executive Chairman of Pillar Biosciences. "In partnership with Illumina, our newest IVD offering, oncoReveal CDx is a pan-cancer solid tumor IVD that will enable critical diagnostic testing to be performed locally in any NGS lab, whether hospital-based or a commercial reference laboratory, running an Illumina MiSeq Dx System. This approach demonstrates our commitment to bringing highly accurate, actionable, and reimbursable NGS testing to clinical laboratories and biopharmaceutical companies to help improve treatment decisions and deliver outcomes that are accessible to everyone, everywhere."

"Illumina is proud to partner with Pillar Biosciences to expand diagnostic offerings on the MiSeq Dx," said Kevin Keegan, General Manager of Oncology at Illumina. "Broadening the market for tumor profiling and therapy selection options is a key enabler of precision medicine and harnesses the power of the genome to improve human health."

The oncoReveal CDx pan-cancer solid tumor IVD, which has been validated and approved for use on the Illumina MiSeq Dx System, was developed for tumor profiling and therapy selection and includes actionable targets on most common solid cancer types. The panel covers 22 clinically relevant genes in one multiplex reaction and has a single-day workflow that can be performed by any clinical laboratory with a sample-to-report time of as little as 48 hours. Up to 46 clinical samples can be batched on a single MiSeq Dx run.

This PMA supplement approval follows the original PMA approval of oncoReveal Dx in July 2021. This IVD is also currently CE IVD approved on MiSeq Dx for EGFR & KRAS for therapy selection in non-small cell lung cancer (NSCLC) and colorectal cancer (CRC), and NMPA approved in China for KRAS, BRAF and PIK3CA for therapy selection in CRC.

On April 23, 2024 Interius BioTherapeutics, a leading developer of in vivo cell-specific gene medicines, reported the presentation of preclinical data across six abstracts at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting to be held on May 7-11 in Baltimore, MD (Press release, Interius BioTherapeutics, APR 23, 2024, View Source [SID1234642254]).

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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

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"Our lead candidate to treat B cell malignancies, INT2104, utilizes Interius’s proprietary lentiviral vector platform that incorporates CD7 targeting to specifically transduce T cells and NK cells upon intravenous (i.v.) administration – without preconditioning chemotherapy," said Phil Johnson, MD, President and CEO of Interius. "Data from humanized mice and cynomolgus macaques demonstrate that a single i.v. administration of INT2104 transduces both T and NK cells that are then reprogramed to become functional chimeric antigen receptor (CAR) cells." Biodistribution and immunohistochemical analyses confirmed vector targeting specificity. The in vivo generated autologous CAR cells targeted CD20 positive cells leading to B cell depletion in both animal models. One macaque had persistent B cell aplasia for one year, and at autopsy, had no detectable B cells in the peripheral circulation, spleen, bone marrow, or lymph nodes.

A formal GLP toxicology study established a substantial and impressive safety profile. In 20 vector-treated cynomolgus macaques, no clinical signs or symptoms of toxicity were observed, and no laboratory abnormalities were recorded over the 180-day study. These data, considered together with an extensive pre-clinical data package, support clinical entry of INT2104 this year for B cell malignancies and the rapid development of the company’s second program for autoimmune diseases.

Details of the presentations are as follows:

Abstract Number and Title: 2. In Vivo Generation of Both CAR T Cells and CAR NK Cells Using a CD7
Targeted Lentiviral Vector
Session: Presidential Symposium
Date and Time: Wednesday, May 8, 11:30-11:45 am
Location: Hall A-B

Abstract Number and Title: 201. Rational Design of a Detargeted Vesiculovirus Fusogen to Enable Targeted In Vivo Gene Delivery
Session: Emerging Viral Vectors
Date and Time: Thursday, May 9, 4:30-4:45 pm
Location: Ballroom 4

Abstract Number and Title: 235. Pharmacokinetics and Vector Shedding in NHPs Following a Single Intravenous Infusion of a CD20-Targeted Engineered Lentiviral Vector
Session: Pharmacology Toxicology Studies and Analytics Assay Development
Date and Time: Friday, May 10, 8:30-8:45 am
Location: Room 339-342

Abstract Number and Title: 1360. Automating a Lentivirus Infectious Titer Assay (ITA)
Poster Session: Vector Product Engineering, Development, and Manufacturing
Date and Time: Thursday, May 9, 12:00 pm – 7:00 pm
Location: Exhibit Hall

Abstract Number and Title: 1361. Evaluation of Residual Host Cell DNA Clearance and Sizing During Production of a Lentiviral Vector
Poster Session: Vector Product Engineering, Development, and Manufacturing,
Date and Time: Thursday, May 9, 12:00 pm – 7:00 pm
Location: Exhibit Hall

Abstract Number and Title: 1839. In Vivo Delivery of an Engineered Lentiviral CAR19 Vector for the Treatment of Autoimmune Diseases
Poster Session: In Vivo Therapy Approaches
Date and Time: Friday, May 10, 12:00 pm – 7:00 pm
Location: Exhibit Hall

On April 23, 2024 Hanmi Pharmaceutical (KOSPI: 128940, CEO: Jae-Hyun Park), a leading biopharma company in Korea that focuses on research areas such as oncology, obesity/metabolism, and rare diseases, reported it has entered into a Clinical Trial Collaboration and Supply Agreement (CTCSA) with MSD (a tradename of Merck & Co., Inc., Rahway, NJ, USA) (Press release, Hanmi, APR 23, 2024, View Source [SID1234642253]).

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Upon the execution of the agreement, Hanmi Pharmaceutical will conduct a phase 1 clinical trial to evaluate the safety and efficacy of its immuno-oncology drug, ‘BH3120’, in combination with MSD’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), in patients with progressive or metastatic solid tumors. Hanmi Pharmaceutical will sponsor the clinical trial, and MSD will supply KEYTRUDA.

‘BH3120’ is a next-generation immunotherapy drug that applies ‘Pentambody’, a bispecific antibody platform technology, currently under joint development by Hanmi Pharmaceutical and its Chinese subsidiary, BJHM (Beijing Hanmi Pharmaceutical).

Pentambody is a technology that combines one antibody to two different targets simultaneously, facilitating both immuno-oncology therapy and targeted therapy.

More specifically, BH3120 is an lgG-like bivalent bispecific antibody targeting PD-L1 and 4-1BB with biased binding affinities towards PD-L1.

This design aims to induce strong anti-tumor activities, particularly in PD-L1 overexpressed tumor tissues within the tumor microenvironment (TME), while minimizing undue immune activation in normal tissues.

Most of the existing antibody candidates targeting 4-1BB have limitations in terms of safety. However, BH3120 has been shown through various non-clinical studies to exhibit a clear decoupling of immune activity between TME and normal tissue, confirming its potential as an effective and safe anticancer agent.

Dr. Kim Dong-wan, the director of the Seoul National University Hospital Clinical Trials Center (Hemato-Oncology Department), is the Principal Investigator of the phase 1 clinical trial of BH3120 in Korea and the US. He said, "Through the collaboration with MSD, we expect BH3120 in combination with KEYTRUDA could improve outcomes for patients with relapsed or refractory disease."

A Hanmi representative stated, "BH3120 is our first global clinical research project using the next-generation bispecific antibody platform technology ‘Pentambody’ in the field of immuno-oncology therapy, potentially changing the paradigm of anticancer treatment," and added, "We are dedicated to surpassing the constraints of current therapies and innovatively boosting therapeutic efficacy through our next-generation immune-oncology therapy."

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

On April 23, 2024 Theriva Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported positive topline data from the investigator sponsored Phase 1 trial conducted by collaborators at Sant Joan de Déu-Barcelona Children’s Hospital (SJD) (Press release, Theriva Biologics, APR 23, 2024, View Source [SID1234642250]). The Phase 1 trial was designed to evaluate the safety and tolerability of two intravitreal injections of Theriva’s investigational oncolytic adenovirus VCN-01 in patients (n=9) with intraocular retinoblastoma that is refractory to chemotherapy or radiotherapy, and for whom enucleation was the only recommended treatment.

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"Results from the investigator sponsored trial further validate VCN-01’s unique mechanism of action and therapeutic potential to improve patient outcomes in otherwise refractory cancers," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "We look forward to building on the encouraging safety profile and antitumor activity, which further supports and informs the design of our proposed Phase 2 clinical trial. The Monitoring Committee determined that the trial results were positive, and therefore, Theriva will receive an exclusive, worldwide license, and related patents from SJD for the treatment of pediatric patients with advanced retinoblastoma. The positive completion of this trial is an important step in refining our clinical strategy for VCN-01 as an adjunct to chemotherapy to address the high unmet need in this underserved indication."

Key Takeaways: Patients received two intravitreal injections of VCN-01, 14 days apart, at a dose of either 2 x 109 vp/eye (n=1) or 2 x 1010 vp/eye (n=8). The data for 9 evaluable patients were reviewed by the study Monitoring Committee who agreed that the trial had a positive outcome:

Safety: VCN-01 was well tolerated after intravitreal administration at the 2 doses and the most frequently reported treatment-related adverse events were Grade 1 or 2. There were no dose limiting toxicities and no ocular or systemic toxicities equal to or greater than Grade 3 during the evaluation period.
Some degree of ocular inflammation and associated turbidity was observed after VCN-01 injection. Inflammation was managed, and vitreous haze improved in some cases, by local and systemic administration of anti-inflammatory drugs.
Antitumor effects: intravitreal VCN-01 demonstrated promising antitumor activity and did not appear to change the retinal function.
Four patients presented a response characterized by unequivocal improvement in vitreous seed density.
Eye enucleation was avoided in 3 patients to date, one of whom has retained their eye after 4 years of follow-up.
About Retinoblastoma

Retinoblastoma is a tumor that originates in the retina and is the most common type of eye cancer in children. It occurs in approximately 1/14,000 – 1/18,000 live newborns and accounts for 15% of the tumors in the pediatric population < 1 year old. The average age of pediatric patients at diagnosis is 2, and it rarely occurs in children older than 6. In the U.S., retinoblastoma shows an incidence rate of 3.3 per 1,000,000 with only about 200 to 300 children diagnosed per year according to the American Cancer Society. Preserving life and preventing the loss of an eye, blindness and other serious effects of treatment that reduce the patient’s life span or the quality of life, remains a challenge. In addition, children with retinoblastoma have been more likely to lose their eye and die of metastatic disease in low-resource countries.

About VCN-01

VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 80 patients in Phase 1 and investigator-sponsored clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.