On April 10, 2023 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported the acceptance of three abstracts at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) taking place from May 16-20, 2023 in Los Angeles, CA (Press release, Abeona Therapeutics, APR 10, 2023, View Source [SID1234629898]). Accepted abstracts include new data on three internally developed investigational preclinical gene therapy product candidates from its adeno-associated virus (AAV) ophthalmology program, including ABO-504 for Stargardt disease, ABO-503 for X-linked retinoschisis (XLRS) and ABO-505 for autosomal dominant optic atrophy (ADOA).

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"At ASGCT (Free ASGCT Whitepaper), the data we present will highlight the potential of our proprietary AAV capsids and gene constructs to express recombinant protein in target eye tissues and rescue mutant phenotypes," said Brian Kevany, Ph.D., Chief Technical Officer and Head of Research at Abeona. "We have started to submit pre-IND meeting requests to the FDA for our gene therapy product candidates and anticipate meetings to take place in the second quarter of 2023. We are excited for the opportunity to share our new data with the scientific community at ASGCT (Free ASGCT Whitepaper)."

Details for the poster presentations are as follows:

Abstract Title: In Vivo Production of Full-Length ABCA4 Protein Following Cre-Mediated Recombination from Dual AAV Vectors in ABCA4-/- Mice
Presenter: Dr. Paul Wille, Director, Product Development, Abeona Therapeutics
Session Date/Time: Wednesday, May 17, 2023 at 12:00 PM PT
Abstract number: 715

Abstract Title: ABO-503, a Novel Gene Therapy for Treatment of X-Linked Retinoschisis
Presenter: Dr. Joseph Fogerty, Senior Scientist, Product Development, Abeona Therapeutics
Session Date/Time: Thursday, May 18, 2023 at 12:00 PM PT
Abstract number: 1131

Abstract Title: AAV Gene Therapy for Autosomal Dominant Optic Atrophy Caused by Mutation in the Opa1 Gene
Presenter: Dr. Rachel Stupay, Senior Scientist, Product Development, Abeona Therapeutics
Session Date/Time: Friday, May 19, 2023 at 12:00 PM PT
Abstract number: 1478

On April 9, 2023 Syncell, a biotech company pioneering in platforms for hypothesis-free spatial proteomics, reported that it will be exhibiting and presenting a poster at the AACR (Free AACR Whitepaper) Annual Meeting 2023 in Orlando, FL. Syncell’s Microscoop technology enables microscopy-guided subcellular protein scooping in ultra-content powered by AI to collect enough proteins for subsequent spatial proteomic identification (Press release, Syncell, APR 9, 2023, View Source [SID1234629896]). Syncell announces that Microscoop Mint, the first commercial Microscoop platform of Syncell, will be available for order. Earlier this year, Syncell also announced expansion of its Global Rapid Access Service Program (GRASP) to use Microscoop.

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"Total-Sync Ultra-Content Microscopic Opto-Biotinylation Enables High-Sensitivity Hypothesis-Free Subcellular Protein Discovery"

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"Syncell’s Microscoop technology is not really a spatial proteomic mapping technology, it is a direct spatial proteomic discovery technology to help researchers identify novel protein biomarkers. We are excited to see the high demands of services for diverse life science problems. We are happy to see recent successful proteomic discovery for customers’ research projects. We believe Microscoop technology will also be broadly useful for cancer research," said Dr. JC Liao, the Founder and CEO of Syncell.

Details of the technology and the new product will be showcased at Syncell’s exhibit hall booth (#129). A poster will be presented on April 18, 2023 @ 1:30 PM – 5:00 PM at Section 23 titled "Total-Sync Ultra-Content Microscopic Opto-Biotinylation Enables High-Sensitivity Hypothesis-Free Subcellular Protein Discovery". Copies of the poster will also be available at the booth.

On April 9, 2023 GC Genome Corporation, a leading genomic diagnostics company, reported the publication of a new study in Radiation Oncology Journal, which demonstrates the feasibility of cell-free DNA(cfDNA) monitoring to predict treatment response and detect minimal residual disease after radiation therapy(RT) in solid tumor patients using I-score, a tool for calculating genomic instabilities developed by GC Genome (Press release, GC Genome, APR 9, 2023, View Source [SID1234629895]). The research was in collaboration with the Samsung Medical Center, Sungkyunkwan University School of Medicine. This study adds to the previous studies of prognostic effects of the I-score in various solid tumors, including hepatocellular carcinoma, esophageal cancer, and pancreatic adenocarcinoma1.2.3.

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The study analyzed 23 plasma samples from cancer patients across a range of lung, esophageal, and head and neck cancer, and plasma samples from 358 healthy people were used as negative controls. Test performance was evaluated at serial cfDNA monitoring points: before RT, 1 week after RT, and 1 month after completion of RT using I-score, which represents chromosomal alterations across the genome(CIN) of cfDNA from Low-coverage whole genome sequencing(LC-WGS) data.

The result showed that pretreatment I-scores tended to be higher in larger tumors, and there was a significant positive correlation between the gross tumor volume and the baseline I-score. Another interesting finding is that minimal residual disease following RT was detected earlier by cfDNA than by imaging studies. And the serial monitoring of the I-score in the post-treatment 4 months case demonstrated that the change in I-score was observed before the progression of the disease was detectable through imaging studies.

"The findings of this study may have significant implications for the field of oncology, particularly for patients with lung, esophageal, and head and neck cancer," said Dr. Chang-Seok Ki, MD, CEO at GC Genome. "Further additional studies are ongoing to optimize the measurement and analysis of I-scores to predict radiation response accurately. The potential of cfDNA I-score as a monitoring tool in cancer treatment deserves continued investigation and attention."

[Reference]

1. Kim EJ, Im HS, Lee J, et al. Genome-wide and size-based cell-free DNA indices as predictive biomarkers for locally advanced esophageal squamous cell carcinoma treated with preoperative or definitive chemoradiotherapy. Curr Probl Cancer 2021;45:100685.

2. Oh CR, Kong SY, Im HS, et al. Genome-wide copy number alteration and VEGFA amplification of circulating cell-free DNA as a biomarker in advanced hepatocellular carcinoma patients treated with Sorafenib. BMC Cancer 2019;19:292.

3. Woo SM, Kim MK, Park B, et al. Genomic instability of circulating tumor DNA as a prognostic marker for pancreatic cancer survival: a prospective cohort study. Cancers (Basel) 2021;13:5466.

On April 7th, 2023, Biosyngen reported it was granted IND approval by China NMPA for the company’s first-in-class T-cell redirection therapy, it is an autologous T cell therapy for EBV-positive lymphoma. The principle of autologous T cell therapy is to genetically modify patients’ own T cells to express additional receptors for Epstein-Barr virus (EBV) antigen recognition and T cell activation upon EBV+ tumor cell engagement, targeting cancer indications (Press release, BioSyngen, APR 7, 2023, View Source;c=View&a=index&aid=97 [SID1234631945]).

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Currently, commercially available cell therapies, such as CD19 CAR-T, are designed for the treatment of B cell lymphoma or acute lymphoblastic leukemia. Treatment options for EBV-associated lymphoproliferative diseases and lymphoma are limited and in urgent demand. The company’s new pipeline targeting EBV-associated lymphoma is a hope for this population of patients. "Biosyngen has been committing itself to high quality R&D of cell therapy. This is the second IND approval obtained for the company’s first-in-class products worldwide, demonstrating our commitment to remain focused on unmet clinical needs of cancer patients," said Dr. Michelle Chen, COO of Biosyngen. "At this point, in addition to this announcement, we have secured IND approvals for nasopharyngeal carcinoma treatment by US FDA and China NMPA. Following closely will be news of Biosyngen’s application to FDA for the treatment of EBV positive lymphoma. In addition, preparation for applications of orphan drug and Fast Track eligibility are also well under way." Biosyngen has continued in building up a pipeline of other cancer indications; within 2023, the company made plans for IND applications for other therapies such as lung cancer and liver cancer across key regions – Singapore, the US and China.

About EBV-positive Lymphoma

EBV, the first oncovirus identified, is a human herpesvirus and has infected ~95% of global population. It has been listed as Group 1 carcinogen ("Carcinogenic to humans") by World Health Organization (WHO) and proved to be associated with a range of diseases including nasopharyngeal cancer, EBV-positive gastric cancers, lymphoma and lymphoproliferative diseases. EBV is so markedly lymphotropic that, for those who suffer from impaired immune system, especially T cell function, EBV may even induce lymphoblastic malignancies. EBV reactivation was also observed in patients who received bone marrow transplantation and CD7 CAR-T cell therapy, who may even develop EBV positive lymphoma.

The therapy developed by Biosyngen is an engineered T cell therapy, also known as a type of adoptive immune cell therapy indicated for nasopharyngeal cancer and EBV-positive lymphoma. Patients’ T cells are isolated and genetically modified in a GMP-compliant facility to enhance their ability to recognize and attack specific antigens on cancer cells. The modified T cells are expanded ex vivo and infused back into the patient. The infused T cells would bind to specific antigen on the cancer cells to mediate tumor killing. The preliminary safety and efficacy of BRG01 Therapy have been demonstrated in data from exploratory clinical trials.

The scientific direction of Biosyngen is focused on targeting multiple solid tumors and hematological tumors. The company has independently developed a number of exclusive technical platforms specifically for cancer immunotherapy, including IDENTIFIER, SUPER-T and MSE-T. These platforms are designed for improved safety and efficacy, equipping the company with capabilities to overcome challenges in antigen identification, antibody TCR screening and identification of immune cell function

On April 7, 2023 PhenomeX Inc, the functional cell biology company, reported its participation at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023 being held from April 14-19 at the Orange County Convention Center in Orlando, Fla (Press release, PhenomeX, APR 7, 2023, View Source [SID1234629892]). AACR (Free AACR Whitepaper) brings together scientists, clinicians, other health care professionals, survivors, patients, and advocates to share the latest advances in cancer science and Medicine."

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At the conference, PhenomeX, the new company recently formed through the combination of Isoplexis and Berkeley Lights, will showcase its IsoSpark and Beacon optofluidic platform technologies and workflows in booth #3444. Attendees will have a chance to explore demonstrations of the technologies and learn more about how PhenomeX’s applications can provide unparalleled insights into cell function along the continuum of scientific discovery, bioprocessing, translational, and clinical research.

In addition, the AACR (Free AACR Whitepaper) Annual Meeting covers the latest advances in cancer through a variety of poster and speaker presentations. This year, PhenomeX technologies are highlighted in nine poster presentations ranging from Polyfunctional Profiles and Cytokine Secretion Activity of Transgenic TCR-T cells and Anti-Cancer Macrophage-Based Cell Therapy to the Identification of Myeloma-Specific T Cell Receptors by Functional Single Cell Interaction Analyses. Some of the presenting abstracts include:

Dr. Scott Nowicki (UCLA) Abstract | Section 37 / 914/29
Dr. Alejandro Villagra (Georgetown) Abstract | Section 37 – 900/15
Dr. Wafik El-Deiry (Brown) Abstract | Section 6 – 2518/26
Dr. Alejandro Villagra (Georgetown) Abstract | Section 21 – 2880/18
UT MD Anderson Abstract | Section 22 | 2903/8
University of Wisconsin, Madison Abstract | Section 25 – 4142/4
National Institutions of Health (NIH) Abstract | Section 23 – 4103/22