Category Archives: Molecular Medicine

BOSTON, Sept. 06, 2022 (GLOBE NEWSWIRE) -- Monte Rosa Therapeutics, Inc. (NASDAQ: GLUE), a biotechnology company developing novel molecular glue degrader (MGD)-based medicines, today announced that the U.S. Food and Drug Administration (FDA) has cleared the companys investigational new drug application (IND) for MRT-2359, a potent and selective GSPT1-directed MGD. The company is on track with initiating a Phase 1/2 clinical trial of MRT-2359 in patients with MYC-driven solid tumors, including lung cancer, and expects to dose the first patient in the fourth quarter of 2022.

The FDAs clearance of our first IND serves as a critical milestone for Monte Rosa and continues to validate our differentiated approach to protein degradation, said Markus Warmuth, M.D., CEO of Monte Rosa. Molecular glue degraders hold tremendous promise in tackling the universe of previously undruggable proteins and fostering a new generation of precision medicine therapeutics. After reporting compelling preclinical data, we are excited to advance MRT-2359 into a Phase 1/2 study for patients with MYC-driven solid tumors who otherwise have limited treatment options.

About MRT-2359MRT-2359 is a potent, selective and orally bioavailable molecular glue degrader (MGD) that induces the interaction between the E3 ubiquitin ligase component cereblon (CRBN) and the translation termination factor GSPT1, leading to the targeted degradation of GSPT1 protein. The MYC transcription factors (cMYC, L-MYC and N-MYC) are well-established drivers of human cancers that maintain high levels of protein translation, which is critical for uncontrolled cell proliferation and tumor growth. Our preclinical studies have shown that this addiction to MYC-induced protein translation creates a dependency on GSPT1. MRT-2359 exploits this vulnerability by inducing degradation of GSPT1, disrupting protein synthesis preferentially in MYC-driven cell lines and leading to anti-tumor activity in MYC-driven tumor models. A Phase 1/2 clinical study aims to evaluate the safety, tolerability and anti-tumor activity of MRT-2359.

About Monte RosaMonte Rosa Therapeutics is a biotechnology company developing a portfolio of novel molecular glue degrader (MGD) medicines. These medicines are designed to employ the bodys natural mechanisms to selectively eliminate therapeutically relevant proteins. The company has developed a proprietary protein degradation platform, called QuEEN(Quantitative andEngineeredElimination ofNeosubstrates), that enables it to rapidly identify protein targets and MGD product candidates that are designed to eliminate therapeutically relevant proteins in a highly selective manner. The companys drug discovery platform combines diverse and proprietary chemical libraries of small molecule protein degraders with in-house proteomics, structural biology, AI/machine learning-based target selection, and computational chemistry capabilities to predict and obtain protein degradation profiles. For more information, visitwww.monterosatx.com.

Forward-Looking StatementsThis communication includes express and implied forward-looking statements, including forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward looking statements include all statements that are not historical facts, and in some cases, can be identified by terms such as may, might, will, could, would, should, expect, intend, plan, objective, anticipate, believe, estimate, predict, potential, continue, ongoing, or the negative of these terms, or other comparable terminology intended to identify statements about the future. Forward-looking statements contained in herein include, but are not limited to, statements about our product development activities, including our expectations around MRT-2359, the ongoing development of our QuEEN platform and the advancement of our pipeline and the various products therein, our expectations of timing, including for initiation and patient dosing, of our clinical trial for MRT-2359, our ability to initiate and the timing of initiation of additional lead optimization programs, and our expectations regarding our ability to nominate and the timing of our nominations of additional development candidates. By their nature, these statements are subject to numerous risks and uncertainties, including the impact that the current COVID-19 pandemic will have on our development activities and operations, as well as those risks and uncertainties set forth in our most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K for the year ended December 31, 2021 filed with the US Securities and Exchange Commission, and any subsequent filings, that could cause actual results, performance or achievement to differ materially and adversely from those anticipated or implied in the statements. You should not rely upon forward looking statements as predictions of future events. Although our management believes that the expectations reflected in our statements are reasonable, we cannot guarantee that the future results, performance or events and circumstances described in the forward-looking statements will be achieved or occur. Recipients are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date such statements are made and should not be construed as statements of fact. We undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, any future presentations or otherwise, except as required by applicable law. Certain information contained in these materials and any statements made orally during any presentation of these materials that relate to the materials or are based on studies, publications, surveys and other data obtained from third-party sources and our own internal estimates and research. While we believe these third-party studies, publications, surveys and other data to be reliable as of the date of these materials, we have not independently verified, and make no representations as to the adequacy, fairness, accuracy or completeness of, any information obtained from third-party sources. In addition, no independent source has evaluated the reasonableness or accuracy of our internal estimates or research and no reliance should be made on any information or statements made in these materials relating to or based on such internal estimates and research.

Contacts:

InvestorsMichael Morabito, Solebury Troutir@monterosatx.com

MediaDan Budwick, 1ABdan@1abmedia.com

See the article here:
Monte Rosa Therapeutics Announces FDA Clearance of Investigational New Drug Application for MRT-2359, a GSPT1-directed Molecular Glue Degrader Phase...

Dr. Anthony Fauci, Bidens chief medical adviser, said in a White House briefing earlier Tuesday: It is becoming increasingly clear that, looking forward with the COVID-19 pandemic, in the absence of a dramatically different variant, we likely are moving towards a path with a vaccination cadence similar to that of the annual influenza vaccine, with annual, updated COVID-19 shots matched to the currently circulating strains for most of the population.

We expect that the updated vaccines will offer better protection against the SARS-CoV-2 subvariants that are currently circulating, Fauci added.

Dr. Ashish Jha, the White House COVID-19 response coordinator, said that barring any new variant curveballs ... for a large majority of Americans, we are moving to a point where a single annual COVID shot should provide a high degree of protection against serious illness all year. Thats an important milestone.

Jha noted that people at highest risk may need more than annual protection, and we will ensure in this administration that they get whatever protection they need.

He also acknowledged the possibility that a new variant could emerge, saying, We plan for what we think is the median, the most likely scenario. But were always watching for that unusual event, and if that happens, we will address it and we will adjust to it and well account for it.

Fauci said there was always the possibility of a wildcard of a way-out, out-of-left-field variant coming in. If that happens, all bets are off, and we change.

Jha said in a stream of tweets Tuesday that the formula to control COVID-19 included the updated vaccines, as well as antiviral medicines, rapid tests, and improved indoor ventilation.

Some experts tweeted that there are still unanswered questions, including how well the updated boosters will work and whether people will get them.

Annual Covid booster? Possibly, but a few stars need to align first I think, tweeted Dr. Peter Hotez, codirector of the Center for Vaccine Development at Texas Childrens Hospital and dean of the National School of Tropical Medicine at Baylor College of Medicine.

Eric Topol, a professor of molecular medicine at Scripps Research, questioned whether the shots would last a whole year, tweeting, I dont see any evidence for how an annual Covid shot will provide durable protection (current ones wane after 4-6 months) without better vaccines. He noted efforts to create a vaccine that will protect against all variants, to improve the lipid nanoparticle delivery system of vaccines, and to develop a nasal vaccine.

Katelyn Jetelina, an epidemiologist in Texas and author of the newsletter Your Local Epidemiologist, said in a post that the administration was gambling because its not clear if an annual vaccination like the flu vaccination will work. The fall bivalent vaccine is ... our first attempt to apply the flu model to SARS-CoV-2. This is our pilot. And we really need to see how the pilot works in the real world before making sweeping declarations, like an annual shot. We need the data, the time, and the humility to tell. Lets first get through winter, she said.

Dr. Robert Wachter, who chairs the Department of Medicine at the University of California at San Francisco, told CNN, The biggest problem with the vaccines today is that people arent getting them.

Wachter said officials were hoping to get more people boosted by taking away uncertainty about when to get shots. The overall goal, he told CNN, was to cast boosters as more manageable, something you do every year, like getting a flu shot.

I think its a really smart way of rebranding and rethinking it, Wachter said.

The Globe reported Sunday it wasnt clear if people were going to get the shots, but doctors said interest might pick up after the holiday weekend and people return to school and work.

Experts say the updated boosters are crucial to blunting the impact of an expected surge of infections this fall and winter.

Staying up to date on vaccines, including boosters, is the most effective way to prevent serious illness, hospitalization, and death from COVID-19. While vaccine protection decreases over time, boosters re-stimulate the immune system and increase vaccine efficacy again. Boosters are an important defense, even if youve already had COVID, the state Department of Public Health said in a statement.

The department said people can check for updates at http://www.mass.gov/covidbooster, and that locations with the updated booster are in the process of being added to vaxfinder.mass.gov.

Winter is not that far away. The past two years, we have seen COVID-19 cases and deaths soar. It does not have to be that way this year. If you are 12 and older, go get your new COVID-19 shot this fall, Biden said in his statement.

Martin Finucane can be reached at martin.finucane@globe.com.

Go here to read the rest:
Biden administration says the once-a-year shot phase of the COVID-19 pandemic has arrived - The Boston Globe

The International Center for Chemical and Biological Sciences (ICCBS), Karachi University held an orientation session for 53 new students MPhil and PhD Program-2022 on Tuesday.

ICCBS director Prof Dr M Iqbal Choudhary welcomed the new students in the Prof Salimuz Zaman Auditorium and asked the newcomers to work hard in pursuance of their higher learning program, as there was no shortcut for scholars in the international center.

Unfolding the importance of sincerity, the ICCBS Director advised the students to adopt sincerity as it was the noblest of all human traits. Sincere people, who are sincere about their work, education, parents, relatives and friends, are capable of securing great success in their life.

He said, The ICCBS, along with its two major wings Dr Panjwani Center for Molecular Medicine and Drug Research, and Hussein Ebrahim Jamal (HEJ) Research Institute of Chemistry, is serving industries and government agencies.

He informed the new students that the center was unique to have the triple honor of UNESCO, WHO and OIC Center of Excellence. This has also received Islamic Development Bank (IsDB) prizes for best science institution in Islamic world, he adds.

Welcoming all the students of the new batch-2022 to the internationally famous doctoral program, Prof Choudhary said that ICCBS owned the single largest 100 percent merit-based doctoral program in the country with over 600 PhD students, including foreign students from different countries of the world.

It is pertinent to mention here that as many as 53 admissions have been given in various disciplines of sciences, which included Organic Chemistry, Analytical Chemistry, Physical Chemistry, Inorganic Chemistry, Bio-Organic Chemistry, Pharmacology, and Molecular Medicine, he added.

Read more:
ICCBS welcomes students of MPhil and PhD Programs 2022 - The Academia Mag

Would you like tocontribute your creativity to an international team of scientistsfrom various disciplines focusing on basic research in the area of molecular life sciences?

The European Molecular Biology Laboratory (EMBL) invites you toapply for PhD positionsin Heidelberg, Barcelona, Grenoble, Hamburg, Hinxton (near Cambridge) and Rome.We welcome candidates with diverse backgrounds, such as in Biology, Chemistry, Physics, Mathematics, Computer Science, Engineering and Molecular Medicine.

Information about the PhD Programme and fellowships as well as research topics at EMBL can be foundhere.

Why join us

EMBL provides PhD students with a starting platform for a successful career in science by fostering early independence and interdisciplinary research. The enriching encounter of different nationalities, the friendly and collaborative atmosphere, and the passion for science is what unites EMBLs diverse staff and provides an ideal setting to forge long-lasting connections and make studying at EMBL a formative experience. Our PhD positions are fully funded and offer broad health care and pension benefits.

Learn more about the EMBL International PhD Programme andapply onlinehere:https://www.embl.org/about/info/embl-international-phd-programme/application/.

Thedeadlinefor submitting the online application is4October 2022. References must be submitted by 6 October 2022.

Interviews will take place in January - February 2023. Successful candidates would start their work at EMBL latest by mid of October 2023.

EMBL is a signatory of DORA. Find out how we implement best practices in research assessment in our recruitment processeshere.

For further information, pleasecontact EMBL Graduate Office viagraduate-office@embl.org.

See the original post:
Exciting PhD positions at the European Molecular Biology Laboratory (EMBL) job with EUROPEAN MOLECULAR BIOLOGY LABORATORY (EMBL) | 308114 - Times...

Dear colleagues and students

Every year the National Research Foundation (NRF) celebrates South African research at its annual awards ceremony. Nominated by their peers and research institutions, awardees are recognised in a variety of categories.

I am extremely proud of the many University of Cape Town (UCT) researchers that were nominated and those that ultimately received accolades at last weeks award ceremony. Six UCT researchers were awarded in three categories.

Professor Rajend Mesthrie (Department of African Studies & Linguistics) received the Hamilton Naki Award to honour the many ways in which he navigated challenges and hardships to deliver an incredibly high standard of research. Professor Mesthrie is one of UCTs A-rated researchers.

Associate Professor Amir Patel (Department of Electrical Engineering) who uses the locomotion of animals to inspire his robotics, and DrWade Petersen (Department of Chemistry) who focuses on the development of new bio-actives, were both awarded the Research Excellence Award for Emerging Researchers for their outstanding research performance.

Jessica Fell (Department of Civil Engineering), Nkosiyomzi Haile Matutu (Department of Psychology) and Athi Welsh (Department of Chemistry) all final-year PhD students were awarded the Research Excellence Award for Next Generation Researchers to celebrate their outstanding academic performances.

What is striking about this years cohort of NRF awardees is that their research has incredible value outside the realm of academia. WhenI read about the focus of their research, it is evident that these are South African researchers with their focus very much on the benefit for South Africa local knowledge to address local challenges through research that is cutting edge at home and globally. This brings our Vision2030 strategy for UCT to life.

UCTs established researchers also shone at this years NRF awards. ProfessorLinda-Gail Bekker (Desmond Tutu HIV Centre) and ProfessorTommie Meyer (Department of Computer Science) improved their ratings to anA. The late Professor Timothy Egan (Department of Chemistry) was also awarded an A-rating posthumously. Emeritus Professor Dirk van Zyl Smit (Centre for Criminology) also achieved a new A-rating.

Additionally, several researchers retained their Aratings, including Professor Anusuya Chinsamy-Turan (Department of Biological Sciences), Professor Valerie Mizrahi (Institute of Infectious Disease and Molecular Medicine), ProfessorGerald Nurick (Department of Mechanical Engineering) and Emeritus Professor David Chidester (Department for the Study of Religions). This brings UCTs researchers who held active Aratings in 2022 to31, the highest of any university in South Africa. ProfessorJess Auerbach (Graduate School of Business) was also awarded a P-rating within one month of joining UCT as a result of support from her previous institution, North-West University.

Please join me in congratulating each of these incredible researchers as well as those colleagues who received a nomination. It is recognition like this that demonstrates that our researchers and research support community are key in securing and retaining our position as the top university in South Africa and on the continent.

My sincere thank you to each of you.

Warm regards

Professor Sue HarrisonDeputy Vice-Chancellor: Research and Internationalisation

Read previous communications:

Read the original here:
Congratulations to our NRF award winners | UCT News - University of Cape Town News

CLEVELANDResearch led by Case Western Reserve University has identified a promising path to developing therapies to treat esophagealtumorsa form ofcancerthat not only is among the most deadly, but also has been occurring at increasing rates over the past decade.

The team of researchers from the School of Medicine and Case Comprehensive Cancer Center (Case CCC) said it has identified a potential therapeutic target for esophageal cancers in the form of a cell signaling pathway known as thetransforming growth factor beta signaling pathway, or TGF-pathway.

This finding is significant because understanding the cell signaling pathwayand associated behaviormay let scientists know what kinds of drug treatments might be effective for a particular cancer. Cell signaling pathways are a series of reactions that begin with a signal from a molecule like a hormone and then continue then down the pathway until the cell function is processed.

The study also linked a protein-coding gene called Hepatocyte nuclear factor 4 alpha (HNF4), to the TGF-pathway, meaning it is a predictive biomarker of cancer. Predictive biomarkers are the key to early cancer detection which is the most important factor when stopping cancer.

This finding is important because it shows us the pathway necessary to target esophageal cancersbut someday maybe other cancers, as well, said Kishore Guda, study senior corresponding author, associate professor at the School of Medicine and member of the Case CCC.

The research teams findings were published recently in the journal Gastroenterology

Understanding treatment options is especially important in instances of esophageal cancer, as approximately 20% of people diagnosed in the United States survive five years, according to the American Cancer Society.

Study implications

Gudas research team discovered in an earlier study that a type of esophageal cancer known as esophageal adenocarcinoma is characterized by the TGF-pathway being hyperactive.

Based on that knowledge and current research from this study, the team reasoned that a new generation drug currently in clinical trials, Vactosertib (ALK5i), which inhibits the TGF-pathway, might be an effective treatment strategy.

From a molecular perspective, our study connects a major transcription factor (HNF4) to a cancer pathway (TGF) that plays fundamental roles in the gastrointestinal tract, Guda said. The knowledge gained from our study could have important implications for other gastrointestinal malignancies such as gastric, pancreatic, hepatic, and colorectal cancers where TGF and HNF4 are highly relevant in disease development.

###

Case Western Reserve University is one of the country's leading private research institutions. Located in Cleveland, we offer a unique combination of forward-thinking educational opportunities in an inspiring cultural setting. Our leading-edge faculty engage in teaching and research in a collaborative, hands-on environment. Our nationally recognized programs include arts and sciences, dental medicine, engineering, law, management, medicine, nursing and social work. About 5,800 undergraduate and 6,300 graduate students comprise our student body. Visitcase.eduto see how Case Western Reserve thinks beyond the possible.

HNF4A Defines Molecular Subtypes and Vulnerability to Transforming Growth Factor -Pathway Targeted Therapies in Cancers of the Distal Esophagus

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.

View original post here:
Patients with some forms of aggressive esophageal cancers may benefit from treatment targeting how cells self-regulate - EurekAlert

The English-language Master's degree programme in Molecular Medicine is directed at highly motivated students from home and abroad who are interested in the subject. The prerequisite is a Bachelor's degree with a focus on either natural sciences or molecular medicine. The degree programme will provide deeper knowledge of topics relating to molecular medicine and clinical theory such as immunology, oncology, genetics, pharmacology and cell biology. In addition, there are also three laboratory internships, each lasting eight weeks. A Master's thesis will be written within six months.

Name of program: Molecular MedicineDegree: Master of Science (M.Sc.)Length of program: 3 semestersProgram start: winter semester onlyApplication deadline: May 30th for winter semester 2021/22 Prerequisites: B.Sc. in Molecular Medicine or a related fieldAdmissions: limited spaces available (apply directly through faculty)Language requirements: English (certification mandatory)

A proof of measles protection is required for enrollment. The form must be completed by your primary care physician.

If necessary, please use the time between your application and enrollment to get vaccinated or to refresh your vaccination status. The short period of time for acceptance after admission is usually not sufficient for this.

For inquiries, please contact the Office of the Dean of Medical Studies studiendekanat@med.uni-goettingen.de.

Program OverviewThe eighteen-month Masters program in Molecular Medicine expands upon the scientific and medical knowledge basis acquired in Bachelor programs and explores in depth the molecular aspects of medicine. Its goal is to train students thoroughly in an area of application of molecular medicine. It should enable students to engage in independent and creative research at the crossroads of medicine and basic science.

Program StructureDuring the first year, students attend the course ?Molecular-pathological aspects of organ systems?, which is designed to heighten their understanding of the interaction between clinical and molecular science. In other obligatory module courses particular aspects of molecular immunology, bacteriology, human genetics and oncology are treated. The final six months are to be devoted to work on a master?s thesis.

A graduate of the Master?s program in Molecular Medicine should have solid knowledge of the methods of scientific research as well as be able to apply a wide spectrum of molecular-medical methods to provide concrete solutions to scientific problems.

Our graduates secure positions in all areas of medical research, laboratory diagnostics, and medical biotechnology, in both the academic and the commercial sectors. Places of employment include: clinics (molecular and biochemical diagnostics, clinical research, etc.); government agencies (criminal investigation offices, public health departments, commercial regulatory authorities, environmental protection agencies, medical associations); places of academic research (universities, Max Planck Institutes and other large research institutions); industrial settings (biomedical technology, production and quality control, basic research and development, publishing, marketing, administration); private laboratories (molecular diagnostics and analysis, environmental protection); and other institutions (ministries, research advancement organizations, technology transfer facilities).

Excerpt from:
Molecular Medicine (M.Sc.) - Georg-August-Universitt Gttingen

Gradalis, Inc.

DALLAS, Aug. 29, 2022 (GLOBE NEWSWIRE) -- Gradalis Inc. announced a peer-reviewed publication today in the Nature portfolio journal, Communications Medicine, in which several biomarkers were evaluated for their potential to predict survival following Vigil (Gemogenovatucel-T) treatment. The publication entitled ENTPD1 as a Predictive Marker of Treatment Response to Gemogenovatucel-T as Maintenance Therapy in Newly Diagnosed Ovarian Cancer, features results from VITAL, a Phase 2b randomized, double-blind, placebo-controlled trial of Vigil in patients with newly diagnosed ovarian cancer. The analysis indicates that pretreatment expression levels of the ENTPD1 gene may be a significant predictor of overall survival (OS) and recurrence-free survival (RFS) following Vigil therapy. Vigil is a novel, personalized cellular immunotherapy platform that is designed to decloak the full repertoire of a patients tumor antigens, reactivate the immune system, and summon key effector cells to deliver a durable clinical response. In VITAL, Vigil showed a positive trend in RFS in the overall population and a significant improvement in RFS and OS in newly diagnosed ovarian cancer patients with BRCAwt and HRP molecular profiles. This finding may allow for a more refined targeting of patients who will benefit from Vigil therapy.

The ENTPD1 gene, also referred to as CD39, is a protein coding gene that functions as a limiting step in the adenosine metabolic pathway found in immunosuppressive tumor microenvironments. ENTPD1 gene directed signaling is involved in a wide range of cancers.In the analysis, a high level of RNA expression by the ENTPD1 gene (High ENTPD1) was prospectively defined as greater than the median value, hence representing 50% of patients. High ENTPD1 was associated with improved RFS and OS following Vigil maintenance treatment in frontline ovarian cancer patients. The analysis was conducted in collaboration with the University of South Alabama.

Story continues

While the use of predictive biomarkers to identify populations most likely to benefit from immunotherapy has expanded and evolved, attempts to use this approach in the treatment of ovarian cancer have thus far been underwhelming, said Rodney Rocconi, M.D., Professor, Obstetrics and Gynecology at University of Alabama at Birmingham and study investigator. The Phase 2b VITAL trial demonstrated statistically significant improvement in RFS and OS when Vigil was used as a frontline maintenance therapy in patients with BRCAwt as well as HRP subtype advanced ovarian cancer. These exciting data and the identification of ENTPD1 as a biomarker that is predictive of response to Vigil, independent of BRCAwt and HRP status, represent a promising step forward with the potential to provide meaningful benefits for ovarian cancer patients.

The full text of the article can be found here: https://www.nature.com/articles/s43856-022-00163-y. Key findings in the paper include:

High ENTPD1 expression predicts response to Vigil versus placebo regardless of HR status: At 40 months elapsed, median RFS was not yet reached in the Vigil treated group (n=23) vs. 8.1 months in the placebo group (n=23), p=0.00007; at 40 months elapsed, median OS was not yet reached in the Vigil treated group vs. 41.4 months in the placebo group, p=0.013.

High ENTPD1 expression with HRP status: RFS and OS were further improved in Vigil patients with tumors demonstrating High ENTPD1 and HRP status compared to the placebo group. Median RFS of 21.1 months was observed in the Vigil treated group (n=11) vs. 5.6 months in the placebo group (n=9), HR=0.18, p=0.004; at 40 months elapsed, median OS was not yet reached in the Vigil treated group vs. 27 months in the placebo group, HR=0.23, p=0.025.

John Nemunaitis, M.D., Chief Scientific Officer of Gradalis commented, ENTPD1 is recognized to be an important gene in cancer development and progression. ENTPD1 has been identified as playing a role in numerous other solid tumor indications, including melanoma, lung cancer and colorectal cancer. There has been growing interest and support from large pharmaceutical companies for the development of drugs targeting the ENTPD1/CD39 axis. The findings published today further underscore the clinical utility of biomarker approaches to predict survival differences across BRCAwt and HRP positive ovarian cancer patients treated with Vigil. We plan to further validate these findings in the Vigil registrational program and hope to use such a biomarker-based patient selection strategy to expand the potential of Vigil across a variety of cancers.

Mr. Steven Engle, Chief Executive Officer of Gradalis added, In 2003, Gradalis founders including our CSO, Dr. Nemunaitis, had the foresight to explore more effective solutions to treat cancer, including approaches that leveraged the patients immune system to target the entire tumor. Based on multiple clinical studies, Gradalis has developed Vigil, an oncology treatment that is designed to decloak the full repertoire of a patients tumor antigens, reactivate the immune system, and summon key effector cells to deliver a durable clinical response. When combined, this powerful trifecta of anti-cancer activity has the potential to eliminate elusive metastatic cells and thus improve survival, as shown in our Phase 2 clinical studies in ovarian cancer.

Mr. Engle continued, Importantly, Vigil activates the patients immune system without disrupting its natural state of balance. As a result, multiple clinical trials have demonstrated that Vigil is well tolerated and has an encouraging safety profile compared to currently approved standard of care treatment options. Taken together, we believe Vigil has the potential to become a game-changing therapeutic approach in oncology. If validated in a planned Phase 3 trial, the ENTPD1 finding may be applicable to multiple cancer tumor types, providing the potential to explore new opportunities to maximize the therapeutic benefit of Vigil.

About Ovarian Cancer and Molecular ProfilesEvery year, an estimated 22,000 patients are diagnosed with ovarian cancer in the U.S. and 14,000 patients die. The biomarker analysis in the VITAL Phase 2b trial focused on patients with ovarian cancer who had the BRCA wildtype (BRCAwt) gene and whose tumors had the homologous recombination proficient (HRP) molecular profile. Patients with the BRCAwt molecular profile have less than a 35% chance of surviving ovarian cancer at five years following diagnosis. BRCAwt is found in over 75% of patients with ovarian cancer and in 90% or more of several other tumor types. Patients with the HRP molecular profile have less than a 30% chance of surviving ovarian cancer at five years following diagnosis. HRP is found in over 40% of patients with ovarian cancer and in 80% or more of several other tumor types, including skin, colorectal and cervical cancers.

BRCA genesproduceproteinsthat help repair damaged DNA and are sometimes calledtumor suppressor genes. There are two types of BRCA genes: BRCA wildtype (BRCAwt) and BRCA mutated (BRCAmt) gene. In tumors of patients with the BRCAwt gene, mutations that lead to the generation of novel neoantigens on the cell membrane of each cell are less likely to occur. As a result, it is easier for the immune system to identify and target the tumor cells. In patients with the BRCAmt gene, mutations are more likely to lead to the generation of different neoantigens on the cell membrane of each new tumor cell making it more difficult for the immune system to identify and target the tumor cells. Vigil uses the patients immune system to target the tumor, so it is not surprising that Vigil would work better in patients with the BRCAwt gene. Similarly, tumors with the HRP molecular profile are more likely to maintain intact DNA repair pathways, and therefore are also more likely to respond to Vigil therapy.

About VigilVigil is a novel, plasmid engineered, autologous tumor cell immunotherapy platform designed to achieve a trifecta of immune anticancer activity using a unique bi-shRNA DNA based technology and the patients own tumor tissue. The trifecta of systemic activity involves knock down of TGF1 and TGF2 which function as tumor suppressor cytokines, increased GM-CSF expression to enhance local immune function and presentation of the patients clonal neoantigen epitopes via use of autologous cancer tissue. By utilizing the patient's own tumor as the antigen source, Vigil is designed to elicit an immune response that is specifically targeted and broadly relevant to each patient's unique clonal tumor neoantigens. Vigil therapy has been well tolerated in Phase 1, 2a and 2b clinical studies.

In VITAL, a multicenter, randomized, double-blind, placebo-controlled Phase 2b trial (NCT02346747), Vigil showed a positive trend in the primary endpoint of recurrence free survival (RFS) in the overall population and a statistically significant improvement in RFS and overall survival (OS), with a median time of three years to date, in a pre-planned subgroup analysis of Stage III/IV newly diagnosed ovarian cancer patients with the BRCAwt molecular profile. In patients with tumors of the HRP type, significant additional improvement was seen in RFS and OS.

Additionally, Phase 1 results in an all-comer clinical trial have shown positive signals of activity in 19 tumor types and some patients treated with Vigil remain in the trial 48 months later. The company is preparing to initiate a clinical trial intended for product registration in patients with the HRP subtype ovarian cancer.

About Gradalis, Inc.Founded in 2003, Gradalis is a privately held, late-stage clinical biotechnology company developing a personalized immunotherapy called Vigil, that has been tested in multiple studies in ovarian and other cancer tumor types. Based on its Phase 2b clinical trial results, the company is preparing to initiate a Phase 3 trial intended for product registration of Vigil in patients with ovarian cancer. Vigil is the first cellular immunotherapy to demonstrate survival benefits in a randomized controlled trial of patients with solid tumors. The results of the companys Phase 2b trial have been published in Lancet Oncology and presented at the American Society of Clinical Oncology. Vigil is being studied in other womens cancer types and has shown positive results in combination with checkpoint inhibitors

Gradalis Vigil platform uses the patients immune system to target the entire tumor. Based on multiple clinical studies, Gradalis has developed an oncology platform that is designed to decloak the full repertoire of a patients tumor antigens, reactivate the immune system, and summon key effector cells to deliver a durable clinical response. When combined, these are a powerful Trifecta of anti-cancer activities, potentially eliminating even the elusive metastatic cells, and as shown in Phase 2 clinical studies in ovarian cancer, a potential gamechanger in oncology. Our clinical trials have also demonstrated that Gradalis platform is better tolerated compared to standard cancer treatments since Vigil uses the patients immune system operating within its natural state of balance rather than in an artificial overdrive as with some technologies. Vigil utilizes proprietary bi-shRNA technology that has been proven to silence multiple genes in a variety of cancers and has the potential to be used in other diseases.

Forward-Looking Statements This press release contains forward-looking statements, including, without limitation, statements regarding the success, cost, and timing of our product development activities and clinical trials, our plans to research, develop, and commercialize our product candidates, and our plans to submit regulatory filings and obtain regulatory approval of our product candidates. These forward-looking statements are based on Gradalis current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks, and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include but are not limited to: (a) the timing, costs, and outcomes of our clinical trials and preclinical studies, (b) the timing and likelihood of regulatory filings and approvals for our product candidates, and (c) the potential market size for our product candidates. These forward-looking statements speak only as of the date made and, other than as required by law, we undertake no obligation to publicly update or revise any forward-looking statements. This press release does not constitute an offer to sell, or a solicitation of an offer to buy, any securities.

Gradalis ContactMark Early(214) 442-8161mearly@gradalisinc.com

LifeSci Advisors ContactJoyce Allaire+1 (617) 435-6602jallaire@lifesciadvisors.com

See the original post:
Gradalis Announces Publication in Nature Communications Medicine Identifying Survival Predicting Biomarker in Patients with Ovarian Cancer Treated...

MANHASSET, N.Y.--(BUSINESS WIRE)--Millions worldwide and an estimated 200,000 people in the United States have lupus, of whom 90 percent are women. In an effort to translate basic molecular research into better therapy for patients seeking care, The Feinstein Institutes for Medical Research has been awarded a $3 million Global Team Science Award from the Lupus Research Alliance. Betty Diamond, MD, director of the Institute of Molecular Medicine at the Feinstein Institutes and the Maureen and Ralph Nappi Professor of Autoimmune Diseases, will lead the research efforts as the corresponding investigator with a multi-disciplinary global team.

Lupus, an autoimmune disease that affects the joints, skin and organs, often causes people to experience fatigue, brain fog, joint pain, rash and fever. There are no cures for the disease; instead, treatments are mostly focused on helping to minimize flare-ups and improve quality of life. Through the support of the award, the project led by Dr. Diamond will aim to characterize the bodys immune and brain response in small numbers of diverse people with Systemic lupus erythematosus (SLE) who are in drug-free remission. Obtaining this information will help paint a better picture of the cells/pathways involved in SLE remission to inform physicians of the pathways to better health.

Some lupus patients can experience reduced flare-ups and go into remission. It is critical to understand what is going on at a molecular level in patients who fall into this category, including patients who still exhibit neuroinflammation. We wonder if those with continuing neuroinflammation are those who will relapse, said Dr. Diamond. Through the generous support of the Lupus Research Alliance, we hope to develop new tools in assessing remission, identifying new signs of lupus flare-ups and novel potential drug targets.

Using brain scans and cell analysis, researchers will help answer questions about which cells are active or inactive during remission, how the brain changes during this time, and other immune response biomarkers that can track the progression of the disease.

As a leader in lupus research, Dr. Diamond brings an important new perspective to unlocking the mysteries of this condition, said Kevin J. Tracey, MD, president and CEO of the Feinstein Institutes and Karches Family Distinguished Chair in Medical Research. Supported by the Lupus Research Alliance, this new project will shed new light on how the brain is interacting with the immune system in these patients.

In addition to Dr. Diamond, members of this global research team include:

This marks the second year the Lupus Research Alliance has awarded its Global Team Science Award. Among other awardees, this year includes international teams led by corresponding investigators; Martin Kriegel, MD, PhD, Head of the Department of Translational Rheumatology and Immunology, University of Mnster, Germany; and Eric Morand, MD, PhD, Head of the School of Clinical Sciences, Monash University, Australia.

Dr. Diamond is a pioneer in the basic science research of autoimmune diseases, particularly SLE. For more than four decades, Dr. Diamond has dedicated her career to the study of DNA-reactive B cells, autoantibodies and their origin and effect on the body. In May 2022, in recognition of her breakthrough achievements in molecular medicine and original research, The National Academy of Sciences elected Dr. Diamond as one of its newest members.

About the Feinstein InstitutesThe Feinstein Institutes for Medical Research is the home of the research institutes of Northwell Health, the largest health care provider and private employer in New York State. Encompassing 50 research labs, 3,000 clinical research studies and 5,000 researchers and staff, the Feinstein Institutes raises the standard of medical innovation through its five institutes of behavioral science, bioelectronic medicine, cancer, health system science, and molecular medicine. We make breakthroughs in genetics, oncology, brain research, mental health, autoimmunity, and are the global scientific leader in bioelectronic medicine a new field of science that has the potential to revolutionize medicine. For more information about how we produce knowledge to cure disease, visit http://feinstein.northwell.edu and follow us on LinkedIn

Read more from the original source:
Feinstein Institutes Get $3M From Lupus Research Alliance to Study Remission and Future Therapies - Business Wire

What is precision medicine?

Precision medicine, also referred to as personalized medicine, is the opposite of a one-treatment-fits-all model, instead tailoring disease prevention, diagnosis and treatment based on a persons own genes or proteins as well as potentially their environment and lifestyle. According to the Precision Medicine Market Report 2022-2023, the increase in scope of application of precision medicine is expected to propel market growth as the leading precision medicine disease focus, oncology, is joined by immunology, central nervous system (CNS), respiratory and other diseases.

Specific to cancer, over the pastseveral decades, researchers, scientists and therapeutic manufacturers in oncology have significantlyimproved treatment outcomes by embracing this precision medicine approach, with some advancements even called revolutionary. Today, cancer patients often undergo molecular tests and genetic profiling to align the physicians treatment selection with the drug most likely to improve a particular patients prognosis. This recognizes that no two cancers are the same, no two patients are the same, and even within established categories, such as breast cancer, different therapeutics will benefit different individuals.

Advances made in oncology are setting a precedent for whats possible in personalized medicine scenarios in other areas and powerfully impacting what the future of tailored care will mean.

Industry visionaries focusing on other disease states and optimizing treatment outcomes are applying the learnings from cancer precision medicine to their own specialties. Rheumatology is one such field. As the prevalence of autoimmune conditions, currently estimated to affect about 3 percent of the US population, increases, it becomes more critical to address individual cases with effective treatments. Like cancer, the underlying factors and disease progression for autoimmune conditions such as lupus, rheumatoid arthritis (RA) and Sjogrens Syndrome vary among those afflicted, and so should the treatment approach.

What makes the promise of precision medicine so appealing to both physicians and patients with autoimmune conditions is that currently, especially for RA, successful treatment response rates to existing therapeutics vary widely and this may be due to physicians taking the one-medication-fits-all approach with first-line therapeutics.

An increase in treatment options for RA has emerged in recent years, particularly when it comesto the development of dozens oftargetedimmunomodulators (TIMs)across five main types of mechanisms of action. However, despite the growing number of TIMs approved to treat RA, drug selection remains a process driven by empirical physician judgment and influenced by insurers drug formulary rules. Phase 3 trials for virtually all RA drugs demonstrate a treatment ceiling whereby approximately 40 percent of patients do not achieve minimal response, and as many as 80 percent do not achieve a major treatment response.

By utilizing a more personalized approach to autoimmune conditions, starting with an accurate diagnosis, physicians can better inform patients and prescribe treatments that hold the most promise for individual symptom relief and in some cases, have the potential to halt disease progression.

Emerging, innovative diagnostic technology promises to change the current trial and errorof assigning treatment protocols for RA, fostering a breakthrough of the treatment response ceiling with drugs that are currently available. Taking a page from oncology, new molecular tests are now a viable option for linking individual patients to the targeted therapy that has the greatest potential for personalized clinical response.

Specific to RA, there are approximately 1.5 million people with the condition in the United States with about 120,000 new diagnoses each year. Targeted biologic therapies in RA are among the largest categories of therapeutic spending in the United States. So, while treatment options are widespread, the key to unlocking the potential of the most effective RA treatment for those affected is in molecular characterization of the synovium the portion of the joints where the drivers of RA damage/progression are present.

These tests, through synovial biopsies, will provide physicians a new approach to the traditional standard of care model. In addition to the obvious benefit of providing more targeted, effective solutions to patients, the tests will also save them time by mitigating repeated trial and error attempts with targeted biologics that leave RA patients without symptom relief while an effective drug is identified. As an example, a study published in Nature Medicine on May 19, 2022, demonstrated that molecular profiling of synovial joint tissue might greatly impact whether certain drug treatments would be effective in treating RA patients.

Furthermore, there is an additional cost benefit: personalized medicine in RA has the potential to reduce billions of dollars spent annually on drugs that do not yield adequate disease control.

Taking a lesson from other disease areas, the potential for innovative diagnostics and a precision medicine approach in treating a variety of autoimmune diseases, including RA, is becoming more and more of a reality and will pave a path to more positive patient outcomes.

Visit link:
Unlocking the Power of Precision Medicine the Rheumatology Example - Technology Networks