Category Archives: Gene Medicine

Janssens Catherine Taylor, vice-president, EMEA medical affairs, therapy area strategy, discusses the importance of systemic innovation across the healthcare system to realise the full value of medicines.

Medicines and vaccines are among the most powerful interventions that can help improve quality of life for people across the world. As an oncology trained medical doctor who practiced in the UK NHS for 10 years, I have seen the benefits first-hand, as patients live longer lives, free from symptoms, and are able to return to work.

Beyond that, there are positive impacts for wider society, healthcare systems and economies. It is essential, therefore, that we continue to streamline the development, regulatory and access process, so that patients have faster access to better and safer medicines.

Cultivating a pro-innovation environment

To achieve this, it will be critical to maintain a pro-innovation environment. Systemic innovation isnt limited to discovering new molecules; its a mindset we must continue to apply across the board. We need to be able to think outside the box and find new interventions and new delivery methods that lead to better and more sustainable standards of medical care.

Because, despite the great improvements made across the industry in recent years to accelerate innovative development, we still face many challenges. Innovative next-generation medicines are met with a series of hurdles such as increased cost of development, regulatory challenges and delays, and longer and more complex clinical trials. And it all fuels the debate: are medicines a cost or an investment?

The answer to this question comes back to our definition of value. Value-based healthcare is a delivery model in which providers are rewarded based on the value and difference they bring to patients lives, rather than volume of treatments provided.

A few months ago, I spoke with Professor Joaquin Mateo, chair of the ESMO Translational Research and Precision Medicine Working Group. We agreed that one of the key future foci of innovation in medicines lay in the treatment of patients with medicines targeted specifically towards the molecular signature of their disease. The ideal state is that one day patients only receive (and the healthcare system only provides) medicines that will improve their condition. While we have made great progress with some diseases, we are not there yet, and we need to ensure that focused investment into research continues to bring the value that patients truly deserve.

The role of innovation in value-based healthcare

Delivering value for patients doesnt start and end in our R&D labs; actions can be implemented across all stages of the drug development lifecycle to encourage and embrace a pro-innovation environment.

For example, alongside stable, transparent and long-term policies or research plans, incentives can encourage innovation from those willing to take risks and tackle areas of unmet patient need. Effective intellectual property systems are also essential in stimulating research and sustainable innovation for the future.

And strong collaboration from all involved in health research is another key component of a value-based healthcare approach. From patients, patient bodies, pharmaceutical companies, and academia, to regulatory bodies, public institutions, and small and medium-sized enterprises (SMEs), all key stakeholders must come together to help the patients who most need new medicines to access them as quickly as possible.

The European Commissions Pharmaceutical Strategy for Europe, adopted in November 2020, outlines a series of concrete actions to ensure accessibility, availability, and affordability of medicines. The Strategy also highlights the importance of drawing lessons from the COVID-19 pandemic to enhance crisis preparedness and response mechanisms, and so help make the healthcare industry more prepared and resilient.

How can innovation within Medical Affairs drive value?

Medical Affairs is at the forefront of driving innovation across our industry, making it an exciting and ever-evolving area to work in. From innovative evidence generation to accelerating medical treatment adoption and transforming medical engagement, our teams play a huge role in helping to deliver much-needed treatments to patients across the world.

According to EFPIA, there are over 7,000 medicines currently in development globally. As part of this, innovative treatments such as CAR-T therapy and retinal gene therapy are emerging as ways to target rare diseases, multiple indications and underserved populations. Such therapies present significant challenges to our current regulatory and access processes, but these are challenges we must overcome if patients are to benefit.

The key, of course, is to always start with the end goal in mind what change do we want to bring to patients? So, alongside developing and providing the treatment itself, its also important to provide comprehensive medical education, and to explore new patient pathways and delivery systems. That way, everyone understands the value of the treatment, and the route to getting it to the right patients at the right time is clear.

Data will always be central to everything we do, as it has the power to communicate the potential of a new treatment to change a persons life. If we are to move towards a value-based healthcare system, then we must utilise the power of both clinical trials data and real-world evidence, to produce the best possible data package for regulatory submissions. Effective data analysis can lead to accelerated access and improved outcomes for patients. And building the voice of the patient into our business innovation is crucial we need patients unique perspectives and experiences to keep us focused on solutions that will have the largest impact on their lives.

At Janssen, we strive to improve access to our innovative medicines and vaccines and achieve the best possible outcomes for patients across the world. Where I feel Medical Affairs can move the needle is as the scientific bridge, engaging with key stakeholders like clinicians, researchers, payers, policymakers, and regulators. Our role is to help translate the data and impactfully articulate the demonstrated medical value of a medicine, and to collaboratively explore how the value of that medicine could be improved. Ultimately, we want to ensure that those patients who could benefit most from our treatments are able to receive them thats what drives our Medical Affairs purpose.

About the author

Between 2016-2019, she was the medical affairs therapeutic area lead in haematology for Janssen Oncology across the EMEA region. Dr Taylor holds an MD from Kings College London and is a member of the UK Royal College of Physicians. Before joining the pharmaceutical industry, she trained and practiced in Clinical Oncology in London for 7 years. She also holds the Diploma in Pharmaceutical Medicine and has completed her UK specialty training in Pharmaceutical Medicine. Prior to joining Janssen in 2015, she held positions in UK, EMEA regional and global medical affairs at Pfizer and Astellas.

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Innovation across the healthcare system is needed to realise the value of medicines - - pharmaphorum

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BostonGene Announces Acceptance of Six Abstracts to be Presented at the American Association for Cancer Research Annual Meeting 2022 - WV News

NEW ORLEANS Researchers at City of Hope, one of the largest cancer research and treatment organizations in the United States, will present late-breaking and clinical trial findings at this years American Association for Cancer Research Annual Meeting, which begins today. These additional presentations showcase a pilot program in tobacco use cessation and new developments in cell therapy.

Engineering natural killer cells for a new type of cell therapy (two presentations)(1) Off-the-shelf cord blood FLT3 CAR-NK cells for immunotherapy of acute myeloid leukemiaPoster Presentation LB102: Monday, April 11, 1:30 to 5 p.m. CT

Jianhua Yu, Michael Caligiuri and colleagues have developed a new cell therapy approach using natural killer cells that, in lab models, induced a greater response against acute myeloid leukemia (AML) and lengthened the survival of mice with AML without damaging healthy blood stem cells. The treatment could one day provide a viable cell therapy option for patients with AML, who traditionally have not benefited from innovative cell therapy treatments. When these patients experience relapse, it often occurs rapidly, so there is not enough time to prepare patient-derived chimeric antigen receptor (CAR) T cell therapy. City of Hope researchers believe the off-the-shelf cord blood approach they are developing will provide a breakthrough treatment option for a subpopulation of AML patients with the FLT3 gene.

(2) Tumor-reactive and anti-PD-L1 co-stimulated killer cells (TRACK-NK) for immunotherapy of non-small cell lung cancerPoster presentation LB211, Wednesday, April 13, 9 a.m. to 12:30 p.m. CT

Ting Lu, Jianhua Yu, Michael Caligiuri and colleagues have engineered off-the-shelf natural killer cells to make a protein that causes them to be 10 times more potent in killing human lung cancer cells grown in the lab. When tested in mice transplanted with human non-small cell lung cancer, the innovative cell therapy City of Hope developed worked better than un-engineered natural killer cells and did not appear to affect body weight, liver or kidney function, or blood counts, suggesting a safe and effective approach to test clinically.

Priming brain tumors with an oncolytic virus before CAR T cell therapy will soon be tested in humansOncolytic viral reshaping of the tumor microenvironment to promote CAR T cell therapy for glioblastomaPoster Presentation CT541, Wednesday, April 13, 9 a.m. to noon CT

Christine Brown will present data to support the initiation of a Phase 1 clinical trial combining CAR T cell therapy with a cancer-killing oncolytic viral therapy for the treatment of recurrent glioblastoma. This combination trial builds on interim clinical findings from City of Hope and the University of Alabama at Birmingham (UAB). A CAR T cell therapy Phase 1 trial being carried out at City of Hope for recurrent glioblastoma suggests that the more immune cells within the tumor, the longer the patients survival. The oncolytic viral clinical Phase 1 trial conducted by UAB used an oncolytic virus engineered for improved gene expression and viral replication to kill specific brain tumor cells; early findings suggest that the virus could activate immune responses in the brain. Based on these human clinical trials, the research team treated mice with brain tumors first with the oncolytic virus, then with cell therapy, and showed that together the two treatments did not cause any side effects. Based on these findings, a clinical trial under a Mustang Bio investigational new drug application will soon open to try this combination on two types of brain tumors.

Empowering cancer patients to design their own smoking cessation program increases desire to quitEmpowering tobacco-using cancer patient initiation of tobacco cessation by a personal pathway to success program during preoperative patient counseling: a feasibility studyPoster Presentation LB553, Friday, April 8, noon to 1 p.m. CT

Cary Presant, Kimlin Ashing, Steven Rosen and colleagues developed a novel Personal Pathway to Success program where cancer patients were able to choose from 27 individualized tobacco cessation services to help them quit smoking prior to surgery. The pilot program was offered to 54 patients in a preoperative anesthesia testing clinic, and 23 completed counseling. The availability of the program increased initial patient interest from less than 10% to more than 50% of patients working to quit smoking. The innovative, personalized intervention program appears to be effective, partially because it reaches cancer patients during presurgery visits, when they seem to be more receptive to a teachable moment of behavioral changes to prevent disease.

# # #

About City of HopeCity of Hope'smission is to deliver the cures of tomorrow to the people who need them today. Founded in 1913, City of Hope has grown into one of the largest cancer research and treatment organizations in the U.S. and one of the leading research centers for diabetes and other life-threatening illnesses. As an independent, NationalCancerInstitute-designated comprehensive cancer center, City of Hope brings a uniquely integrated model to patients, spanning cancer care, research and development, academics and training, and innovation initiatives. Research and technology developed at City of Hope has been the basis fornumerous breakthrough cancer medicines, as well as human synthetic insulin and monoclonal antibodies. A leader inbone marrow transplantationand immunotherapy, such asCAR T cell therapy, City of Hopes personalized treatment protocols help advance cancer care throughout the world.

With a goal of expanding access to the latest discoveries and leading-edge care to more patients, families and communities, City of Hopes growing national system includes its main Los Angeles campus, a network of clinical care locations across Southern California, a new cancer center in Orange County, California, scheduled to open in 2022, andCancer Treatment Centers of America. City of Hopes affiliated family of organizations includesTranslational Genomics Research InstituteandAccessHopeTM. For more information aboutCity of Hope, follow us onFacebook,Twitter,YouTube,InstagramandLinkedIn.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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City of Hope to present late-breaking and clinical trial data at AACR: Cell therapy and preventive medicine - EurekAlert

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By Loretta Piccenna 08 April 2022

Neuroscience researchers have foundgenes in common involved in dysregulation in brain tissue samples of people with Alzheimers Disease (AD) and temporal lobe epilepsy (TLE).

People living with Alzheimers Disease (AD) have an increased risk of developing seizures, in fact a 10-fold risk which grows higher in people with early onset or familial AD. Conversely, people with epilepsy have a high risk of developing dementia*, suggesting a bidirectional relationship between the two neurological diseases.

Researchers from the Kwan group within the Department of Neuroscience have found two synaptic signalling-associated modules driven by the SCN3B and EPHA4 genes and and two neurogenesis-associated modules driven by the GABRB3 and SCN2A to be dysregulated in brain tissue samples of people with Alzheimers Disease (AD), temporal lobe epilepsy (TLE), indicating similar rewiring that may be involved in the molecular mechanisms.

Electrical abnormalities (epileptiform activity) occur in the temporal brain regions in people with AD. Temporal lobe epilepsy is a common subtype of epilepsy that results in focal seizures and has a similar pathophysiology with AD (e.g. tau pathology, amyloid deposition and hippocampal sclerosis). However, the underlying pathophysiology and causal relationships between the two diseases are poorly understood.

Weighted Gene Coexpression Network Analysis (WGCNA) and network preservation statistics methods were applied to gene networks for temporal lobe epilepsy, AD and controls (see schema above).

Dr Alison Anderson, senior author of the study said, What we found were specific defects in molecular mechanisms that are shared between the two neurological diseases. This finding is particularly important as it may uncover the cause of how epilepsy develops in AD and open up new doorways for more targeted treatment options for patients.

Anna Harutyunyan, a PhD student (Jones Group), said, As first author, together with seniorauthor Dr Alison Anderson, we are among the very few women (probably <10%) that have contributed to the journals topic where our study was published - Advanced computational strategies to tackle human complex diseases. The team were also the only Australian group who have published their research in this topic.

Reference

Harutyunyan, A, Jones, N.C., Kwan, P, Anderson, A. Network Preservation Analysis Reveals Dysregulated Synaptic Modules and Regulatory Hubs Shared Between Alzheimers Disease and Temporal Lobe Epilepsy. Frontiers in Genetics. 2022; 13,https://doi.org/10.3389/fgene.2022.821343.

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*Central Clinical School's Consumer and Researcher Engagement (CARE) Committee is hosting a webinar on dementia on 18 May at 5.30 pm - see details and register

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Link between Alzheimer's Disease and epilepsy explored - Medicine, Nursing and Health Sciences - Monash University

"The preclinical studies that we are presenting at AACR 2022 provide a window into five innovative programs in Antengene's pipeline," said Bo Shan, Ph.D., Chief Scientific Officer of Antengene. "These programs target areas that we believe are very important in cancer drug development: Tumor microenvironment (TME) regulators (ATG-037), pathway inhibitors (ATG-018, ATG-022 and ATG-008), and ADCs (ATG-022). These studies have been instrumental in guiding our clinical development plans for each program, including selection of combination partners and biomarkers, that could be used to predict efficacy or improve the proportion of patients who respond to treatment. We are very pleased to share these results with the oncology community."

Details of the posters and corresponding abstracts are shown below:

ATG-037, a highly potent small molecule CD73 inhibitor has superior activity of reversing immunosuppression in higher-AMP environments compared with anti-CD73 antibodies

Abstract:2576

Session:Cell Cycle, Replication Inhibitors, and Immunotherapy Agents

Date and Time:9:00 AM 12:30 PM CST, April 12, 2022

Venue:Poster Section 21

This study was designed to compare the T-cell rescue activity of ATG-037, a highly potent and selective oral small molecule inhibitor of CD73, and two CD73 blocking antibodies. CD73 is an enzyme that is highly expressed in the tumor microenvironment and enables the degradation of AMP into adenosine, resulting in immunosuppression and cancer progression. In vitro assays were used to assess each compound's ability to inhibit CD73 enzyme activity and reverse AMP/adenosine mediated T-cell suppression. ATG-037 demonstrated more potent and complete inhibitory activity of cell surface CD73 in this study.

As shown in Figure, the authors found that ATG-037 had a stronger ability to restore T-cell function in higher-AMP environments compared with other clinical anti-CD73 antibodies. These data highlight the potential therapeutic advantages of small molecule inhibitors of CD73 over blocking antibodies. ATG-037 is being evaluated by Antengene in a Phase I trialasmonotherapy and in combination with anti-PD-1 antibody in patients with locally advanced or metastatic solid tumors.

The novel ATR inhibitor ATG-018 is efficacious in preclinical cancer models

Abstract:2604

Session:DNA Damage Response and Repair

Date and Time:9:00 AM 12:30 PM CST, April 12, 2022

Venue:Poster Section 22

In this study, the preclinicalpharmacology data set supporting the development of ATG-018, a small molecule ATR inhibitor, was reviewed. Inhibiting ATR kinase (ataxia telangiectasia and Rad3 related kinase) leads to increased accumulation of double-strand breaks, particularly meaningful for tumor cells which rely on DNA damage response (DDR). ATG-018 was tested in a panel of 142tumor cell lines and three CDX mouse models to assess anti-tumor efficacy and to identify potential predictive biomarkers. ATG-018 was a potent inhibitor of in vitro ATR activity inhibition and cell proliferation without significant impact on normalperipheral blood mononuclear cell (PBMCs) viability.

In addition, a series of genetic alterations were discovered that correlated with ATG-018 sensitivity and could be potential predictive biomarkers. As shown in Figure, the authors found that ATG-018 demonstrated potent in vivo efficacy in solid tumor/hematologic cancer models with certain DDR-related mutations. These data showed the potential of ATG-018 in synthetic lethality with homologous recombination deficiencies and promising application in a wide range of indications.With single-agent activity and no impact on PBMCs viability, ATG-018 may be well positioned for use in mono- or combination therapy in a wide range of tumors that rely on DDR. Development of a set of predictive biomarkers could enable its use as a precision-medicine. Antengene intends to file the first IND for ATG-018 in 2022.

ATG-022, an antibody-drug conjugate targeting Claudin 18.2, demonstrated potent in vivo efficacy in gastric cancer patient-derived xenografts

Abstract:1143

Session:Preclinical and Clinical Pharmacology

Date and Time:9:00 AM 12:30 PM CST, April 11, 2022

Venue:Poster Section 25

In this preclinical study, ATG-022, an antibody-drug conjugate targeting Claudin18.2 (CLDN18.2), was evaluated in several gastric cancer patient-derived xenograft (PDX) models, to assess whether it had potential across a range of CLDN18.2 expression levels. Human CLDN18.2 is ectopically expressed in a large number of gastric and pancreatic cancers. Monoclonalantibody targeting CLDN18.2 demonstrated a promising clinical benefit when used in combination with chemotherapy. However, it showed suboptimal efficacy in patients with low CLDN18.2 levels.

In this study presented in the AACR, ATG-022 is reported to show high affinity (sub-nanomolar grade) against CLDN18.2 and demonstrated potent in vitro andin vivoantitumor effects, within vivoefficacy observed in CLDN18.2 low expression PDX models. As shown in Figure, ATG-022 demonstrated much better in vivo efficacy compared with benchmark ADC.In addition, ATG-022 was highly specific for CLDN18.2, with virtually no effect on cells expressing CLDN18.1. In addition, ATG-022 has almost no impact on body weight, a proxy for safety. The authors concluded that ATG-022 shows promise for treating gastric cancer patients with a broad range of CLDN18.2 expression levels, a significant unmet need. Antengene is conducting preclinical studies for ATG-022.

Synergistic effects of the combination of Kras (G12C) with SHP2, ERK 1/2, mTORC1/2 or XPO1 inhibition for the treatment of Kras (G12C) mutated cancer

Abstract:2679

Session:Signaling Pathway Inhibitors

Date and Time:9:00 AM 12:30 PM CST, April 12, 2022

Venue:Poster Section 25

This preclinical study was conducted to identify combination therapy regimen that could overcome the short progression free survival that is a characteristic of KRAS G12C inhibitors (linked to acquired resistance). The study evaluated the anti-tumor activity of ATG-012, a KRAS G12C inhibitor, with four other agents that are involved in the multiple pathways impacted by RASi: i) an SHP2 inhibitor (ET0038), ii) an ERK 1/2 kinase inhibitor (ATG-017), iii) an mTORC1/2 kinase inhibitor (ATG-008) or iv) the XPO-1 inhibitor, Selinexor, in preclinical solid tumor CDX models.

While ATG-012 monotherapy induced dose-dependent tumor growth inhibition at day 27, as shown in Figure, the authors also found strong in vivo synergism in 2-agent combinations. In particular, ATG-012 and clinical stage ERK inhibitor (ATG-017) demonstrate strong in vitro and in vivo synergism, suggesting potential clinical application which may overcome the rapid resistance of KRAS inhibitors. These data open the door to a range of combination partners for ATG-012 that could be fine-tuned to address drug resistance and potentially improve progression-free survival by matching tumor type/histology and combination partner for patients with the KRAS G12C mutation. Antengene is conducting preclinical studies for ATG-012.

Identification of MUC5B mutation as a positive predictive biomarker for mTORC1/2 inhibition by ATG-008 in lung cancer

Abstract:4032

Session:Molecular Pharmacology

Date and Time:9:00 AM 12:30 PM CST, April 13, 2022

Venue:Poster Section 26

This study was designed to evaluate whether MUC5B could serve as a positive predictive biomarker for mTORC1/2 inhibition by ATG-008 (Onatasertib) in lung cancer. ATG-008 is a dual mTOR complex 1/2 kinase inhibitor. The mTOR complex regulates cell growth, metabolism, proliferation and survival. While the mTOR pathway is frequently deregulated in cancers, efficacy of mTOR inhibitors in lung cancer has been modest. In the study, 31 lung cancer cell lines were treated with ATG-008 to determine dose response and to correlate the gene mutation, amplification and expression with sensitivity to ATG-008.

As shown in Figure, the authors found that the presence of the MUC5B mutation correlates with more potent anti-tumor efficacy of ATG-008 in vitro and in vivo in lung cancer CDX models. The mucin MUC5B has a critical protective role in normal lung and has been identified as prognostic marker in multiple tumor types. One observation highlighted in the poster is that MUC5B is also mutated in melanoma, endometrial, colorectal, esophogastric and cervical cancers, vastly expanding the potential clinical utility of MUC5B mutation as the predictive biomarker for ATG-008.ATG-008 is being evaluated by Antengene in multiple Phase I andII clinical trials.

About Antengene

Antengene Corporation Limited ("Antengene", SEHK: 6996.HK) is a leading commercial-stage R&D-driven global biopharmaceutical company focused on innovative first-in-class/best-in-class therapeutic medicines for cancer and other life-threatening diseases. Driven by its vision of "Treating Patients Beyond Borders", Antengene aims to provide the most advanced anti-cancer drugs to patients in the Asia Pacific Region and around the world. Since initiating operations in 2017, Antengene has obtained 23 investigational new drug (IND) approvals in the US and in Asia, submitted 6 new drug applications (NDAs) in multiple Asia Pacific markets, with the NDA for selinexor/ATG-010/XPOVIO in China, South Korea, Singapore and Australia approved. Leveraging partnerships as well as in-house drug discovery, Antengene has built a broad and expanding pipeline of 15 clinical and pre-clinical assets. Antengene has global rights on 10 programs and Asia Pacific rights, including the Greater China region, on 5 programs.

Forward-looking statements

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development. For a further discussion of these and other factors that could cause future results to differ materially from any forward-looking statement, see the section titled "Risk Factors" in our periodic reports filed with the Hong Kong Stock Exchange and the other risks and uncertainties described in the Company's Annual Report for year-end December 31, 2020, and subsequent filings with the Hong Kong Stock Exchange.

For more information, please contact:

Investor Contacts:

Donald LungE-mail: [emailprotected] Mobile: +86 18420672158

PR Contacts:

Peter QianE-mail: [emailprotected]Mobile: +86 13062747000

SOURCE Antengene Corporation Limited

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Antengene Announces Publication of Five Posters at the 2022 American Association for Cancer Research (AACR) Annual Meeting - PR Newswire

Data to be presented at the 2022 AACR annual meeting highlights the unique nature of post-chemotherapy derived gene expression signatures in stratifying patient outcomes

IRVING, Texas, April 8, 2022 /PRNewswire/ -- Caris Life Sciences(Caris), the leading molecular science and technology company actively developing and delivering innovative solutions to revolutionize healthcare, to present findings that provide a deeper understanding that tumor expression of genes related to extent of drug exposure, stratified by p53 status, is associated with clinical outcomes on the common chemotherapeutic regimens used to treat metastatic colorectal cancer (CRC). These results will be presented at the 2022 American Association for Cancer Research (AACR) Annual Meeting being held April 8 - 13, 2022 in New Orleans, Louisiana.

The research with poster titled, "Prognostic and predictive drug-induced gene signatures for colorectal cancer patients personalized based on p53 status and treatment with FOLFOX, 5-FU, oxaliplatin or irinotecan" (Abstract #1231), was led by Wafik El-Deiry, M.D., Ph.D., FACP, Director of Brown University's Legorreta Cancer Center, Associate Dean at the Warren Alpert Medical School, a member of Caris' Precision Oncology Alliance (POA). Caris' POA is a growing network of leading cancer centers across the globe that collaborate to advance precision oncology and biomarker-driven research. This work is being presented in New Orleans by Lindsey Carlsen, a Pathobiology graduate student in the EL-DEIRY Lab at Brown.

The goal of this study was to identify predictive biomarkers for chemotherapies used in CRC. The study used CRC cell lines to identify differentially expressed genes following 5-fluorouracil, irinotecan, or oxaliplatin treatment and stratified the signatures based on p53 status. From these in vitro studies, the researchers then examined whether these genes and gene signatures could predict CRC patient outcomes following chemotherapy (FOLFOX, 5-fluorouracil, irinotecan or oxaliplatin). 2,983 wild-type and 6,229 loss-of-function p53 CRC patient samples were analyzed by DNA/RNA next-generation sequencing at Caris Life Sciences. Real-world survival outcomes were inferred from insurance claims data and Kaplan-Meier estimates. Both prognostic and non-prognostic gene expression had a significant effect on survival outcomes following specific drug treatments.

"This study helps us understand the importance that gene signatures have in demonstrating an enhanced predictive ability compared to individual transcripts," said El-Deiry."Bridging basic and clinical research, this research allows us to better understand which therapies are more likely to benefit CRC patients." The study found that tumor expression of genes related to drug exposure can predict outcomes after chemotherapy treatment:

"Caris is focused on biomarkers that can predict patient response to the newest therapies, but also tried and true chemotherapies like FOLFOX," said W. Michael Korn,M.D., Chief Medical Officer at Caris. "We are excited to see the translational research capability empowered by the tremendous multi-omic data and analytical tools that Caris has developed and continues to make progress on."

Caris' comprehensive molecular profiling assesses whole exome (DNA), whole transcriptome (RNA) and protein expression, providing an unmatched resource and the ideal path forward to conduct the translational research to accelerate discovery for detection, diagnosis, monitoring, therapy selection and drug development to improve the human condition.

Caris will present additional data from studies demonstrating the critical role of precision medicine and molecular profiling in the treatment of cancer. All presentations will be made available online through Caris' website beginning April 8, 2022.

Additional Presentations Reveal Impact of Comprehensive Molecular Profiling and Potential Clinical Actionability

About Caris Life SciencesCaris Life Sciences(Caris) is the leading molecular science and technology company actively developing and delivering innovative solutions to revolutionize healthcare and improve patient outcomes. Through comprehensive molecular profiling (Whole Exome and Whole Transcriptome Sequencing) and the application of advanced artificial intelligence (AI) and machine learning algorithms, Caris has created the large-scale clinico-genomic database and cognitive computing needed to analyze and unravel the molecular complexity of disease. This information provides an unmatched resource and the ideal path forward to conduct the basic, fundamental research to accelerate discovery for detection, diagnosis, monitoring, therapy selection and drug development to improve the human condition.

With a primary focus on cancer, Caris' suite of market-leading molecular profiling offerings assesses DNA, RNA and proteins to reveal a molecular blueprint that helps patients, physicians and researchers better detect, diagnose and treat patients. Caris' latest advancement, which is currently available within its Precision Oncology Alliance, is a blood-based, circulating nucleic acids sequencing (cNAS) assay that combines comprehensive molecular analysis (Whole Exome and Whole Transcriptome Sequencing from blood) and serial monitoring making it the most powerful liquid biopsy assay ever developed.

Headquartered in Irving, Texas, Caris has offices in Phoenix, New York, Denver, Tokyo, Japan and Basel, Switzerland. Caris provides services throughout the U.S., Europe, Asia and other international markets. To learn more, please visitCarisLifeSciences.comor follow us on Twitter (@CarisLS).

Caris Life Sciences Media Contact: Ann ObeneyVice President, Corporate Communications[emailprotected]469-550-1589

SOURCE Caris Life Sciences

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New Research from the Caris Precision Oncology Alliance Finds Prognostic and Predictive Drug-Induced Gene Signatures for Colorectal Cancer Patients -...

Neferteri Strickland

On Thursday, April 21, 2022, from 1:30 2:30 pm at Alfred Ely Beach High School, twenty-five Medical Explorer Post students will participate in CRISPR Classrooms Meet a Scientist program. In so doing, the Explorers will gain (1) an improved awareness of biotechnology careers, (2) a better understanding of the current possibilities and the problems in the industry, (3) a deeper appreciation for the real power of the scientific method.

Alfred Ely Beach High School alumna, Neferteri Strickland is sponsoring and funding this ground-breaking event for the Medical Explorers through her STEM Orga- nization, TEACHERS&, A Benefit LLC. This group of Medical Explorers from the Memorial Health Medical Explorer Post and the A. E. Beach High School Medical Explorer Post will be the first high school students in Georgia to be introduced to CRISPR. Neferteri explained why CRISPR is a valuable tool in STEM education and what inspired her to bring this presentation to students at her high school alma mater, Genomic technologies like gene editing promise to improve the treatment of illness and disease through precision medicine versus traditional therapies. As the future workforce in this field, todays youth should learn about these new career pathways as early as possible.

For more information about this event or to arrange an interview, contact Barbara Foley at 912- 308-7892 or barbara.foley@scouting.org.

CRISPR is a genetic biotechnology that allows scientists to rewrite DNA; CRISPR offers hope for a new wave of strategies to cure diseases, solve food-shortage crises, and even help mitigate climate change. CRISPR Classroom is an education technology company that works to ensure students, and people in general, have access to learn about gene editing and associated biotechnologies. CRISPR Classroom develops turnkey biotechnology content for educators, aligned with Next Generation Science Standards, to promote critical thinking, communication, and collaboration in high school and college students. Additionally, by connecting learners with real scientists and biotechnology professionals through our Meet a Scientist program, we humanize the science industry in a way that supports connected engagement. To learn how you can bring CRISPR Classroom programs and scientists to your students email us at hello@crisprclassroom.org or visit our website at http://www.crisprclassroom.org.

Dr. Kristina Tatiossian, Ph.D., a professional CRISPR scientist and founder of CRISPR Classroom, will virtually meet the Explorers to discuss gene editing and help them understand how CRISPR works, Having received her B.S. in Entomology from UC Davis (Davis, CA) and her Ph.D. in Medical Biology from the University of Southern California (Los Angeles, CA), she has made a career out of deeply understanding the magical biological world around and within us. You can find Dr. Tatiossian, on LinkedIn http://www.linkedin.com/in/tatiossian/ where she invites all interested in her journey to connect and say hello.

Founder of TEACHERS&, Neferteri Strickland is a 1997 graduate of Alfred Ely Beach High School. She is currently a Communications Officer with the Army National Guard, Adjunct Professor of Blockchain Technology at St. Josephs University and Cybertechnology Strategist. TEACHERS& is an education consultancy designed to support education stakeholders as they navigate the future of education. Learn more about their education focused entrepreneur support network at https:// http://www.teachersand.com/

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Savannah Students First In Georgia To Be Introduced To CRISPR & Gene Editing Technology Careers - The Savannah Tribune - Savannah Tribune

Parkinsons disease is a chronic neurological disorder that comes with no cure or definitive prevention techniques.

Photo : iStock

New Delhi: Parkinsons disease is a neurological movement disorder charactertised by tremours, stiffness, shaking, balance, and coordination problems. A disease with no cure, this condition begins slowly and then progresses over time. Both men and women can develop this condition; however, men are 50 per cent more prone to it. Age is often cited as the main risk factor associated with Parkinsons with the majority of patients developing it at 60 or after; yet, there are some patients who may have to struggle with an early onset that may happen before 50. This World Parkinsons Day, we bring to you a detailed account of the neural disorder, its most debilitating symptoms, and a few coping tips for patients.

What causes Parkinsons?

What are the symptoms of Parkinsons?

Parkinsons is the second-most-common neurodegenerative disease that affects adults globally. In India in 2016, 0.58 million people were reported living with Parkinsons. As per the Global Burden of Disease study in 2018, the prevalence of Parkinsons disease has doubled globally over the last two decades.

Parkinsons usually begins slowly and then gets worse over time. Some of the early and most common symptoms that patients struggle with are:

Some patients may also report behavioural changes like:

How to cope with Parkinsons disease?

Parkinsons disease is a chronic neurological disorder that comes with no cure or definitive prevention techniques. Yet, there are some simple non-medicine ways of dealing with it.

Disclaimer: Tips and suggestions mentioned in the article are for general information purposes only and should not be construed as professional medical advice. Always consult your doctor or a dietician before starting any fitness programme or making any changes to your diet.

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World Parkinson's Day: Debilitating symptoms that most patients struggle with; coping tips based on latest res - Times Now

Mental health disorders including depression, schizophrenia, and anorexia show links to biological markers detected in routine blood tests, according to our new study of genetic, biochemical and psychiatric data from almost a million people.

The research will increase our understanding of what causes mental illness and may help to identify new treatments.

People often consider mental health as separate from the health of the rest of the body. This is far from true: there is clear evidence many biochemical substances involved in diseases such as diabetes and autoimmune conditions directly impact the function of our brain.

Many studies have tried to address this by focusing on substances called biomarkers that can be readily measured in blood.

A biomarker is simply something in the body that is a sign of a particular disease or process. These often relate to the kind of things reported in a blood test ordered by your doctor, such as cholesterol, blood sugar, liver enzymes, vitamins, or markers of inflammation.

Biomarkers found in routine blood tests are useful as they are often affected by diet and lifestyle, or by treatment with a drug.

Its often difficult to study the role of these blood biomarkers in mental health conditions. Many studies in this area are often not large enough to make strong conclusions.

One solution is to look at genetic influences on both mental illness and substances measured in blood. Genetics is useful as we now have data from millions of individuals who have volunteered in research studies.

Both mental illnesses and blood biomarkers are what geneticists call complex traits. In complex traits, many genes are involved and environmental factors also contribute.

Read more: Genetics helps estimate the risk of disease but how much does it really tell us?

The widespread availability of genetic data has allowed us to investigate how huge numbers of tiny changes in the DNA sequence (or variants) are related to the risk of mental illness. These same variants can then also be linked to the measured levels of a biomarker in blood.

For example, a variant in a particular gene may increase the risk of developing schizophrenia and also be linked to a decrease in the levels of a vitamin circulating in the blood. Most of these variants are individually associated with very small changes in something like risk of a mental illness, but they may add together to produce larger effects.

Our recent study sought to use genetics to investigate the relationship between nine mental health disorders and 50 factors measured in routine blood tests, such as cholesterol, vitamins, enzymes, and indicators of inflammation. We used data from very large studies conducted by other people, with data from almost a million volunteers included in total.

Our study first confirmed the existence of what is called genetic correlation between blood biomarkers and mental illness, which was more widespread than has previously been shown. Genetic correlation means the effect of DNA sequence changes on the risk of a mental illness and the levels of a given biomarker were more similar to each other than what would occur by chance alone.

To take one example, there was a positive genetic correlation in our study between white blood cell count and depression. This could indicate some process in our body influences both depression and white blood cells.

If we could identify what this shared process is, it could lead to a better understanding of what causes depression and this could be targeted for treatment.

Our study showed there was correlation between the genetics of mental illness and factors in the blood, but this does not tell us whether blood biomarkers are involved in what causes mental illness.

To distinguish correlation from causation in medicine, the gold standard approach is to conduct clinical trials where patients randomly receive a treatment or a placebo. However, these trials are expensive and difficult to conduct.

We did the next best thing: using DNA variants linked to changes in blood biomarkers to act as a natural clinical trial. This process takes advantage of the fact we randomly inherit DNA variants from our parents, in much the same way that participants in a clinical trial randomly receive a treatment or a placebo.

Its a complex method and the results need careful interpretation.

We found evidence some substances measured in blood may actually be involved in the cause of some mental illnesses. Proteins related to the immune system, for example, may be involved in depression, schizophrenia, and anorexia.

Further work is now needed to identify how these blood measures are precisely involved in these disorders, and to find out if they can be targeted for treatment.

Read more: Blame it on biology: how explanations of mental illness influence treatment

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We found a genetic link between routine blood test results and mental health disorders - The Conversation

Akouos, Inc.

- Advanced toward planned IND submissions for AK-OTOF in the first half of 2022 and AK-antiVEGF in 2022

- Presented data at ARO demonstrating potential of precision genetic medicine platform to address a broad range of inner ear conditions

- Expanded leadership team with appointment of otology leader Aaron Tward, M.D., Ph.D., as chief scientific officer

- Ended 2021 with a strong cash position of $232.5 million

BOSTON, March 29, 2022 (GLOBE NEWSWIRE) -- Akouos, Inc. (Nasdaq: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today reports financial results for the fourth quarter and full year ended December 31, 2021 and provides business highlights.

In 2021, we continued to execute on our core strategic objectives, ended the year with a strong cash position, and remained focused on our IND submission plans for AK-OTOF in the first half of 2022 and for AK-antiVEGF in 2022. In addition to AK-OTOF and AK-antiVEGF, we continue to leverage our genetic medicine platform to expand our pipeline and address a broader range of inner ear conditions, including some of the most common forms of hearing loss, said Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos. We recently presented nonclinical data at ARO that help support these future development plans, and we expanded our world-class team of experts with the appointment of renowned surgeon and scientist Aaron Tward, M.D., Ph.D., as our chief scientific officer. We believe the genetic medicines we are developing have the potential to create a new standard of care for, and to transform the lives of individuals and families with, disabling hearing loss.

Pipeline and Business Highlights

Continued progress toward IND submission readiness for lead programs AK-OTOF and AK-antiVEGF Akouos continued to advance its lead product candidate, AK-OTOF, a gene therapy intended for the treatment of otoferlin gene (OTOF)-mediated hearing loss and is on track to submit an investigational new drug (IND) application in the first half of 2022. Additionally, Akouos continues to plan for an IND submission in 2022 for AK-antiVEGF, a gene therapy candidate in preclinical development for the potential treatment of patients with vestibular schwannoma.

Applying genetic medicines platform to expand into broader range of inner ear conditions Akouos is leveraging its multimodal genetic medicine capabilities to address a broad range of inner ear conditions, including those that are monogenic and those of complex etiology. The company recently presented nonclinical data at the Association for Research in Otolaryngology (ARO) 45th Annual Mid-Winter Meeting that help support future development of gene therapies targeting inner ear conditions.

Two nonclinical studies in non-human primates evaluating protein expression and tolerability support future clinical development of AK-antiVEGF for the treatment of vestibular schwannoma.

AAVAnc80 with a supporting cell selective promoter drives widespread GJB2 expression in supporting cells, while limiting expression in, and loss of, hair cells in mice. We continue to evaluate the most promising product candidate options in mice and non-human primates.

In vitro characterization of AAV-mediated RNA interference gene silencing and CRISPR/Cas9 gene editing methods demonstrates a reduction of protein expression for the gene of interest. We continue to consider targets for autosomal dominant nonsyndromic hearing loss.

We also continue to believe that AAVAnc gene therapy has the potential to restore hearing in individuals with a wide range of environmental hearing loss by regenerating hair cells from neighboring supporting cells. We have identified multiple factors that, when delivered in combination, result in new hair cell formation in neonate mice, and we plan to continue preclinical development work in 2022.

Expanded leadership team with appointment of Aaron Tward, M.D., Ph.D., as chief scientific officer In March 2022, Akouos announced the appointment of surgeon and scientist Aaron Tward, M.D., Ph.D., as chief scientific officer. Dr. Tward brings deep experience in genetics, genomics, gene delivery, high-throughput sequencing technologies, and the clinical care of patients with conditions of the ear and skull base to Akouos. Dr. Tward was previously a member of the Akouos scientific advisory board since 2018. In his new role as chief scientific officer, he will lead the research team and provide strategic scientific expertise to advance the companys precision genetic medicine platform.

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Fourth Quarter and Full Year 2021 Financial Results

Cash Position Cash, cash equivalents, and marketable securities were $232.5 million as of December 31, 2021, as compared to $308.0 million as of December 31, 2020.

Research and Development (R&D) Expenses R&D expenses were $18.8 million for the fourth quarter of 2021 and $64.6 million for the full year ended December 31, 2021, compared to $8.0 million for the fourth quarter of 2020 and $34.3 million for the full year ended December 31, 2020. The increase was primarily due to the increased efforts in IND-enabling studies and increased manufacturing costs for AK-OTOF and AK-antiVEGF and the growth in the number of R&D employees and their related activities, as well as the expense allocated to R&D related to Akouoss leased facilities.

General and Administrative (G&A) Expenses G&A expenses were $6.2 million for the fourth quarter of 2021 and $22.2 million for the full year ended December 31, 2021, compared to $4.6 million for the fourth quarter of 2020 and $14.6 million for the full year ended December 31, 2020. The increase was due to the growth in the number of G&A employees and other administrative expenses related to operating as a public company, as well as the expense allocated to G&A related to Akouoss leased facilities and due to increased patent activities and increases in professional fees related to legal and accounting services.

Net Loss Net loss was $24.9 million, or $0.72 per share, for the fourth quarter of 2021 and $86.7 million, or $2.52 per share, for the full year ended December 31, 2021, compared to $12.5 million, or $0.37 per share, for the fourth quarter of 2020 and $48.6 million, or $2.77 per share, for the full year ended December 31, 2020.

About Akouos

Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

Forward-Looking Statements

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, and timing of our future clinical trials and our research and development programs, and the timing of our IND submissions for AK-OTOF and AK-antiVEGF. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our limited operating history; uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether results from nonclinical studies will be predictive of results or success of clinical trials; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing, and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations and any changes in such laws and regulations; risks related to competitive programs; the potential that our internal manufacturing capabilities and/or external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the Risk Factors included in the Companys Quarterly Report on Form 10-Q for the three months ended September 30, 2021 filed with the Securities and Exchange Commission on November 9, 2021, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Condensed Consolidated Balance Sheet Data (Unaudited)

(in thousands)

December 31, 2021

December 31, 2020

Cash, cash equivalents and marketable securities

$

232,452

$

308,010

Total assets

278,755

333,350

Total liabilities

45,105

22,736

Total stockholders equity

233,650

310,614

Condensed Consolidated Statements of Operations and Comprehensive Loss(Unaudited)

(in thousands, except share and per share data)

Three Months Ended December 31,

Years Ended December 31,

2021

2020

2021

2020

Operating expenses:

Research and development

$

18,819

$

7,977

$

64,595

$

34,297

General and administrative

6,158

4,646

22,226

14,583

Total operating expenses

24,977

12,623

86,821

48,880

Loss from operations

(24,977

)

(12,623

)

(86,821

)

(48,880

)

Other income (expense):

Interest income

326

366

1,872

567

Other expense, net

(288

)

(291

)

(1,722

)

(287

)

Total other income, net

38

75

150

280

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Akouos Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Business Highlights - Yahoo Finance