On November 17, 2025 CorriXR Therapeutics, Inc., an oncology-focused biotherapeutics company pioneering a novel gene editing platform to overcome drug resistance in solid tumors, reported the publication of a manuscript in Molecular Therapy Oncology detailing results from a preclinical study evaluating CRISPR-directed gene editing for the treatment of squamous cell lung carcinoma (LUSC). The study was conducted in collaboration with scientists at ChristianaCare’s Gene Editing Institute (GEI).

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"This foundational work strengthens CorriXR’s strategy of disrupting cancer cell survival pathways to restore sensitivity to standard therapies," said Eric B. Kmiec, Ph.D., Founder and Chief Executive Officer of CorriXR Therapeutics and Executive Director of GEI. "These findings build on more than a decade of GEI research into NRF2, a master regulator of cellular stress responses and known driver of treatment resistance. We are encouraged by the consistency of results across in vitro human lung cancer models and our in vivo studies and are actively pursuing IND-enabling work to bring this promising approach to patients."

Key findings from the study include:

Restoration of chemosensitivity: Editing 20-40% of LUSC cells to disrupt NRF2 was sufficient to resensitize tumors to chemotherapy, resulting in significant reductions in tumor growth.
Reduced cancer-driving signals: Edited tumors reduced NRF2 expression and downregulation of its downstream markers, demonstrating effective pathway disruption.
No off-target editing above background: Unintended edits remained below 0.2% supporting the specificity and safety of the gene editing approach.
Strong translational potential: The lipid nanoparticle (LNP) delivery system achieved robust editing in both engineered and patient-derived tumor models, reinforcing the feasibility of advancing towards clinical development.
"Treatment resistance remains one of the greatest challenges in oncology, and these data demonstrate that targeting NRF2 can meaningfully resensitize tumors with minimal off-target effects," said Kelly Banas, Ph.D., lead author of the study and Associate Director of Research at GEI. "This approach has the potential to lower chemotherapy doses, reduce toxicity and help patients remain healthier throughout treatment." Kmiec added, "Instead of creating entirely new drugs, we are using gene editing to make existing ones effective again."

The study also highlights that the biology of NRF2 driven resistance extends beyond lung cancer. "While this work focused on LUSC, NRF2 overactivation drives treatment resistance across multiple solid tumors, including head and neck squamous cell carcinoma (HNSCC)," said Kmiec. "These data indicate that CRISPR-enabled targeting of NRF2 may disrupt the tumor microenvironment and address a shared mechanism of therapeutic failure."

LUSC is an aggressive form of non-small cell lung cancer (NSCLC), representing 20-30% of lung cancer cases and affecting an estimated 190,000 people annually in the U.S. Chemotherapy remains a cornerstone of care, but many patients develop resistance, leaving limited options beyond dose escalation, which increases toxicity and typically worsens quality of life. NRF2 overactivation is a well-established driver of this resistance across multiple solid tumors, including HNSCC, esophageal and liver cancers – representing a significant unmet medical need.

These findings provide a compelling foundation to advance CorriXR’s lead program for HNSCC, as well as the Company’s LUSC program, into clinical development. CorriXR and GEI are now independently validating results at commercial CROs, conducting the required safety and regulatory studies to support an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for approval of human trials, and are exploring partnerships to accelerate clinical translation.

The full publication: Functional characterization of tumor-specific CRISPR-directed gene editing as a combinatorial therapy for the treatment of solid tumors – ScienceDirect is available online in Molecular Therapy Oncology.

(Press release, CorriXR Therapeutics, NOV 17, 2025, View Source [SID1234660040])

On November 17, 2025 Acuitas Therapeutics, a global leader in lipid nanoparticle (LNP) delivery systems for the acceleration of partners’ clinical development, reported its Next-Generation LNP advancements, a suite of novel and enhanced technologies that expand the range of diseases treatable with mRNA-LNP medicines, at the 13th International mRNA Health Conference in Berlin. Also at the conference, the company highlighted additional preclinical data on its LNP formulations’ applicability in cancer vaccines, potency, and safety.

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"Commercial validation of our technology has provided both an important milestone of success and the impetus for continued advancement," said Dr. Thomas Madden, CEO of Acuitas Therapeutics. "Our research, presented at the mRNA Health Conference, is focused on two key goals: expanding the application of our technology into new therapeutic areas and enhancing the potency and safety of the platform itself, with novel LNP formulations and various improvement strategies. Underpinning both is our commitment to improving the translatability of preclinical data, which is essential for accelerating the journey of mRNA and personalized therapies from the laboratory bench to the clinic."

Next-Generation LNP for mRNA-based Therapeutics

At the conference, Acuitas’ Chief Scientific Officer, Dr. Ying Tam, showcased the company’s Next-Generation LNP advancements, a comprehensive approach that uses multiple technologies and strategies to improve all aspects of LNP utility and applicability – from potency, safety, extrahepatic targeting, to manufacturing.

The advancements were featured in an oral presentation titled "Next-Generation Lipid Nanoparticles for Clinical Development of mRNA-based Therapeutics." The presentation detailed Acuitas’ efforts in advancing mRNA-LNP beyond current clinical standards and broadening the range of diseases these therapies can treat. Key highlights of the presentation include:

Novel LNP candidates engineered for significantly improved potency, as high as a four-fold increase for some cases, in gene editing and vaccine applications.
Optimized lipid structures reduced liver exposure, leading to increased tolerability, lowered liver enzymes, while preserving therapeutic activity in mice.
DARPin-conjugated LNP candidates that achieved highly targeted delivery to immune cells (T-lymphocytes), with a long-circulating format further enhancing uptake efficiency and expression levels.
Mucous penetrant mRNA-LNP candidates capable of extrahepatic delivery to airway epithelial cells in cystic fibrosis lung models, enabling effective gene editing compared to control LNP.
An alternative LNP manufacturing approach, called pre-formed vesicles (PFV), with equivalent potency to standard benchmark manufacturing methods, offered significant improvements in cost, storage, distribution, and flexibility of LNP manufacturing, especially for personalized mRNA-LNP therapies.
Additional Posters Presented

In addition to its lead presentation, Acuitas also showcased three posters that elucidated the mechanics of LNP delivery and assessed its existing slate of lipids, as well as explored novel options for future formulations.

Applying Clinically Approved ALC-315 in Cancer Vaccines

Acuitas’ ALC-315 ionizable lipid — used in the Pfizer-BioNTech COVID-19 vaccine (COMIRNATY) — was assessed for its cancer vaccine development potential. The research directly compared Acuitas’ LNP to lipoplexes and evaluated modified mRNA against the unmodified mRNA predominantly used in current cancer vaccine trials. Key highlights of this data include:

Using LNP comprised of ALC-315, unmodified RNA induced a stronger antigen-specific CD8 T-cell response compared to a nucleoside-modified mRNA incorporating N1methylpseudouridine-encoding OVA payload as a model tumour antigen, while maintaining strong immunogenicity at one-tenth of the initially tested dose.
Using LNP comprised of ALC-315, mRNA delivered intramuscularly induced equal or superior cellular and humoral immunity compared to intravenously (IV) administered mRNA lipoplexes – an alternative mRNA cancer vaccine format in clinical development – despite mRNA lipoplexes being administered at four-fold higher doses and with more boosts.
Several potent novel proprietary lipids were identified that achieved equivalent cellular responses to ALC-315. Further assessment will be conducted to compare potency and activity using syngeneic neoantigen models.
Novel Lipids with Improved Activity for Prophylactic Vaccine Development

Acuitas identified and validated six novel lipids that induce higher virus-specific immunogenicity compared to ALC-315. Key findings related to these novel lipids include:

The six novel lipid candidates induced equivalent neutralizing antibody titres at a five-fold lower dose than ALC-315.
The lipid candidates demonstrated favorable reactogenicity profiles comparable to ALC-315, while eliciting stronger cellular- and B-cell responses.
Innate immune responses induced by LNP correlated with their reactogenicity, but not with adaptive and innate immune responses.
Several lipid candidates achieved higher in vivo expression in secondary lymphoid organs and reduced liver expression compared to ALC-315.
Impact of Body Weight and Medications on mRNA-LNP Safety in Monkeys

As the industry continues using larger nonhuman primates in translational work, Acuitas sought to understand how body weight, premedications (steroid, H1 and H2 blockers), and concomitant medications (meloxicam) impact LNP activity and tolerability. The study was conducted in monkeys using an IV-administered mRNA-LNP encoding human IgG. Key highlights of this data include:

LNP tolerability is reduced in larger monkeys (>6 kg).
Premedications helped reduce the elevation of liver transaminases.
Premedications improved tolerability but reduced the level of IgG mRNA expression.
Platelet count decreases were greatest in large monkeys and monkeys given meloxicam.
More information on posters presented at the mRNA Health Conference and Vaccine R&D Conference can be found here.

(Press release, Acuitas Therapeutics, NOV 17, 2025, View Source [SID1234660039])

On November 17, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that the International Nonproprietary Names (INN) Expert Committee of the World Health Organization (WHO) approved "liraltagene autoleucel" for the non-proprietary name of the Company’s novel FSHR-targeted CAR-T therapy for recurrent ovarian cancer. The INN nomenclature scheme for CAR-T cell therapies follows a two-word structure describing the gene and cell component.

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"The assignment of the non-proprietary name represents an important step in the development and potential future commercialization of our CAR-T therapy. The INN naming process meticulously evaluates proposed drug names for adherence to nomenclature guidelines and potential conflicts, followed by expert consensus and public review. With this INN approval, we have the ability to establish a universally recognized and conflict-free non-proprietary drug name for our CAR-T therapy," said Dr. Amit Kumar, Chairman and CEO of Anixa. "Looking ahead, we remain focused on the successful execution of our ongoing Phase 1 trial of liraltagene autoleucel for the treatment of ovarian cancer, and look forward to commencing the 5th dose cohort in the coming weeks. The Phase 1 study is being conducted in partnership with Moffitt Cancer Center."

Each INN name is unique and is used to identify active pharmaceutical ingredients. Each active substance that is to be marketed as a pharmaceutical must be granted a unique name of worldwide acceptability to ensure the clear identification, safe prescription and dispensing of medicines to patients. Anixa will transition to the use of liraltagene autoleucel, or lira-cel, in future communications.

Jose R. Conejo-Garcia, M.D., Ph.D., Professor of Immunology in the Department of Integrative Immunobiology at the Duke University School of Medicine and the co-inventor of Anixa’s CAR-T technology, stated, "It is gratifying to see this technology advance under Anixa’s guidance and through the work being performed at Moffitt Cancer Center. Receiving its non-proprietary name from the WHO is an exciting step in the process toward commercialization of this technology."

About liraltagene autoleucel
Liraltagene autoleucel, or lira-cel, is a follicle stimulating hormone receptor (FSHR)-mediated chimeric antigen receptor-T cell (CAR-T) technology that targets FSHR, which is exclusively expressed on normal ovarian cells, tumor vasculature, and certain cancer cells. Since the target is a hormone (chimeric endocrine) receptor, and the target-binding domain is derived from its natural ligand, this technology is also known as CER-T (chimeric endocrine receptor-T cell) therapy, a new type of CAR-T. Liraltagene autoleucel is currently being evaluated in a first-in-human trial (NCT05316129) that is enrolling adult women with recurrent ovarian cancer who have progressed after at least two prior therapies. The study is designed to evaluate safety, identify the maximum tolerated dose, and monitor clinical activity. Lira-cel is based on technology exclusively licensed to Anixa by The Wistar Institute.

(Press release, Anixa Biosciences, NOV 17, 2025, View Source [SID1234660038])

On November 17, 2025 Accession Therapeutics Limited, a biopharmaceutical company developing next generation cancer immunotherapies, reported it has dosed the first patient in its Phase I clinical trial evaluating TROCEPT-01 (ATTR-01), the company’s lead drug candidate from its proprietary TROCEPT platform technology. The patient has now completed the cycle of treatment. This milestone marks a major step forward in the development of a potentially transformative treatment for solid tumours.

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TROCEPT-01 (ATTR-01) is a first-in-class, tumour-activated viral immunotherapy that, following systemic delivery, is designed to selectively generate a clinically validated checkpoint inhibitor within tumours. This approach, targeting αvβ6 integrin on epithelial tumours, optimises efficacy while minimising damage to healthy tissue. Preclinical studies have demonstrated strong anti-tumour activity across multiple solid tumour models, supporting its potential as a targeted therapy for aggressive cancers.

The ATTEST trial is an open label, dose-escalation and dose expansion study (View Source) designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of TROCEPT-01 (ATTR-01) in patients with advanced carcinomas who have had at least one prior treatment course. The trial is being conducted at leading clinical sites across the UK. Additional trial sites will be activated as the study progresses, including in Spain where there is established expertise in the treatment of solid tumours.

Professor Adel Samson, Professor of Cancer Medicine and Immunotherapy at Leeds University School of Medicine and lead Investigator in the ATTEST study, said:
"As a clinician, I am very excited about the potential of TROCEPT-01 (ATTR-01) and the TROCEPT platform to increase clinical response rates through high tumour-localised production of anti-cancer drugs. This program is targeted at solid tumours where there is significant need for better treatment outcomes".

Bent Jakobsen, PhD FMedSci, CEO of Accession Therapeutics, said:
"Dosing the first patient in our TROCEPT-01 clinical trial is a pivotal moment for Accession Therapeutics and a testament to the dedication of our team and collaborators. In TROCEPT, we have created a unique, highly versatile platform that enables novel drugs to be made inside cancer cells. The platform gives us multiple opportunities to generate valuable products to transform outcomes for cancer patients. TROCEPT-01 (ATTR-01) has the potential to expand the indications where checkpoint inhibitors have been successful."

Professor Hardev Pandha, FRCP, FRACP, PhD, Medical Director of Accession Therapeutics, added:
"TROCEPT-01 (ATTR-01) represents a novel approach to target hard-to-treat cancers, and we are excited to advance this promising therapy into clinical development. We anticipate the clinical data will show that the virus gets to the tumours when given systemically, and that the drug is produced within the tumours. Our goal in the ATTEST study is to establish a strong safety profile while identifying early signals of efficacy to benefit patients with limited treatment options."

(Press release, Accession Therapeutics, NOV 17, 2025, View Source [SID1234660037])

On November 17, 2025 Biotheryx, Inc., a biopharmaceutical company focused on the discovery and development of first-in-class protein degraders for cancer and inflammatory diseases, reported the completion of enrollment in the ongoing Phase 1a clinical trial of BTX-9341, a potent and selective CDK4/6 degrader, for the treatment of advanced and/or metastatic HR+/HER2- breast cancer in patients who have previously received CDK4/6 inhibitor therapy either in the adjuvant or metastatic setting.

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The Phase 1a clinical trial began with dose escalation of BTX-9341 as a monotherapy, followed by a combination with fulvestrant. The primary objective of the Phase 1a trial is to assess safety, tolerability, pharmacokinetic and pharmacodynamic activity of BTX-9341 as a monotherapy and in combination with fulvestrant. Based on the recommended dose from Phase 1a, there will be a formal evaluation of efficacy in the dose expansion phase of the trial.

"Completing enrollment in the Phase 1a trial for BTX-9341 marks a significant step forward in advancing a very promising first-in-class treatment option for patients with HR+/HER2- breast cancer who have received prior CDK4/6 inhibitor therapy." said Dr. Leah Fung, Chief Executive Officer of Biotheryx. "We are deeply grateful to the patients, investigators, and our team who made this possible as we continue to work together towards the common goal of improving patient lives."

About BTX-9341

BTX-9341 is a first-in-class, oral degrader of CDK4/6, important targets for a range of cancers and clinically validated in HR+/HER2- breast cancer. In preclinical breast cancer models, BTX-9341 demonstrated superiority to CDK4/6 inhibitors through potent and highly selective catalytic degradation of CDK4 and CDK6, robust inhibition of Cyclin E and CDK2 transcription, cell cycle arrest and ultimately superior in vivo efficacy in breast cancer xenografts. Beyond this increased efficacy potential, BTX-9341 is differentiated from CDK4/6 inhibitor approaches through the ability to overcome key resistance mechanisms that limit the impact of inhibitors in second line HR+/HER2- breast cancer.

(Press release, BioTheryX, NOV 17, 2025, View Source;breast-cancer-302616179.html [SID1234660036])