Category Archives: Gene Medicine

REDWOOD CITY, Calif., Sept. 23, 2022 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. ADVM, a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today announced that Peter Soparkar, chief operating officer of Adverum Biotechnologies, will present at the Jefferies Cell and Genetic Medicine Summit on September 29, 2022, at 9:30 a.m. ET.

The on-demand webcast corporate presentation may be accessed underEvents and Presentationsin the Investors section of Adverum's website. A replay of the webcast will be available on the website for 90 days following the presentation.

About Adverum Biotechnologies

Adverum Biotechnologies ADVM is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing functional cures to restore vision and prevent blindness. Leveraging the research capabilities of its proprietary, intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians' offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. By overcoming the challenges associated with current treatment paradigms for these debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe. For more information, please visitwww.adverum.com.

Corporate & Investor Inquiries

Anand ReddiVice President, Head of Corporate Strategy, External Affairs and EngagementAdverum Biotechnologies, Inc.T: 650-649-1358E:areddi@adverum.com

Media

Megan TalonAssociate Director, Corporate CommunicationsAdverum Biotechnologies, Inc.T: 650-649-1006E:mtalon@adverum.com

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Adverum Biotechnologies to Participate in the Jefferies Cell and Genetic Medicine Summit - Adverum Biotec - Benzinga

Every person is unique, from their fingerprints to their DNA. Why then would a one-size-fits-all treatment program be appropriate for every person with a particular illness or condition?

Thats the basic idea behind the precision medicine, or personalized medicine, movement.

Precision medicine is a means of providing health care tailored to a patients individual characteristics, right down to the genetic level, says Dr. Antony Ruggeri, hematology and oncology physician at Aurora St. Lukes Medical Center.

When devising a targeted treatment, precision medicine considers not only a persons environment and lifestyle, but also their DNA. Each persons DNA is made up of unique gene patterns and variations that control their bodys functions.

In a sense, doctors have been personalizing medicine for years, Dr. Ruggeri says. We might not treat a middle-aged patient the same as an elderly patient. But where precision medicine has more recently taken a monumental step forward is in the use of molecular testing to determine a treatment course based on genetic makeup.

Modern molecular testing allows doctors to sequence, or identify, large portions of a persons DNA and then recommend a specific treatment based on a persons specific genetic variations.

Recent advances in precision medicine have led to powerful changes in disease treatment, particularly in the field of cancer care.

Cancer researchers have found that individual tumors also have unique molecular footprints, explains Dr. Ruggeri. Even among the same type of cancer, the genetic changes driving tumor growth will vary.

By taking a tumor tissue sample and comparing the tumors genetic makeup to those of other tumors recorded in an electronic database, doctors may find a treatment with a history of success against a tumor thats genetically similar.

For example, the American Society of Clinical Oncology Targeted Agent and Profiling Utilization Registry (TAPUR) study now underway at Advocate Aurora Health cancer clinics across Wisconsin is evaluating precision medicine cancer treatments with dozens of anticancer drugs that are already on the market.

The drugs available through the study are all approved by the U.S. Food and Drug Administration (FDA) for the treatment of a type of cancer but havent been FDA-approved to treat each study participants specific type of cancer. Researchers hope the study will help identify new treatments for many different types of advanced cancer.

Want to know more about research at Advocate Aurora Health? Visit aah.org/research.

ASCO, American Society of Clinical Oncology, and TAPUR are trademarks of the American Society of Clinical Oncology, Inc., used with permission.

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What exactly is precision medicine anyway? - health enews

Weill Cornell Medicine investigators have identified definitive biological links between African ancestry and disease processes that affect an aggressive cancer type called triple-negative breast cancer (TNBC). Their analysis of TNBC tumors from a diverse patient population yielded a large set of genes whose expression differed in patients with African ancestry compared with patients with European ancestry.

In the study, published Sept. 19 in Cancer Discovery, a journal of the American Association for Cancer Research, the scientists identify the expression of 613 genes associated with African ancestry and more than 2,000 genes associated with regional African ancestry in patients with TNBC. They also describe distinct patterns of immune responses in patients of African descent that may explain patterns of disease progression and outcomes. Together, these findings provide a foundation for future research into better treatment options for this cancer, which has the worst survival outcomes of all breast cancer types.

Many people are not aware of the geographic origins of their ancestors nor how much of their DNA was inherited from each source, known as genetic ancestry. Previous studies of racial differences in TNBC analyzed data from African American patients and relied on self-reported race, said senior author Dr. Melissa B. Davis, associate professor of cell and developmental biology research in surgery and director of health equity in the Englander Institute for Precision Medicine at Weill Cornell Medicine. Our study is the first to determine each individuals ancestry not only by African descent but also by specific regions within Africa.

TNBC tumor cells have no estrogen or progesterone receptors and scant amounts of HER2/neu protein on their surface, making them challenging to treat as they dont respond to hormone therapies or anti-HER2 drugs that block cell proliferation. The subtype represents about 33 percent of breast cancer diagnoses in African countries compared with less than 20 percent in other nations. African American women have twice the risk of developing TNBC and a higher risk of mortality than white Americans of European ancestry.

For their current study, the investigators performed ancestry estimation on breast tissue samples from 132 patients and RNA sequencing on a subset of 26 cases provided by the Englander Institute of Precision Medicine at Weill Cornell Medicine; the University of Alabama at Birmingham; and The International Center for the Study of Breast Cancer Subtypes (ICSBCS), now headquartered at Weill Cornell Medicine. The ICSBCS was established in 2004 and features partners across different regions of Africa as well as the Caribbean and Central America. This study drew samples from ICSBCS founding member The Komfo Anokye Teaching Hospital (KATH) in Kumasi, Ghana, as well as the Millennium Medical College St. Pauls Hospital in Addis Ababa, Ethiopia.

After identifying the expression of genes associated with African ancestry at the country and regional levels, the researchers examined the affected biological pathways and estimated proportions of immune cells in tumors. They discovered that women with TNBC with a high degree of African ancestry, primarily East Africans from Ethiopia, had significantly higher immune cell populations infiltrating tumors, than women with a lower degree of African ancestry who were mainly African Americans and West Africans from Ghana. Increased immune responses in TNBC tumors in women of regional African descent will be particularly interesting to researchers studying the benefits of immunotherapies, said lead author Dr. Rachel Martini, a postdoctoral associate in surgery at Weill Cornell Medicine.

This recent discovery gives us hope that we will continue to find answers and contribute to solutions for a disease which has long afflicted all ancestries, but shows greater burden in Africa, said Dr. Ernest Adjei, consulting pathologist at KATH. The ICSBCS provides a great platform for strong research collaborations into the future as we work together for improved outcomes in breast cancer management.

The investigators also found that several African ancestry-associated genes detected in normal breast tissue switched expressions in tumor tissue. These findings suggest that some ancestry-specific differences in gene expression may be in response to malignancies, said Dr. Martini.

Finally, the researchers examined the data by self-reported race and found some of the same pathways they had associated with ancestry. However, they also found others imprinted on tumors relating to diabetes and obesity that were not associated with ancestry. This finding suggests its essential to look at both race and ancestry when exploring disparities in TNBC development and outcomes, said Dr. Davis, who is an ethnicity scholar at the New York Genome Center and also serves as scientific director of ICSBCS. For example, we could potentially harness aspects of the diabetes or obesity pathways in tumors as targets to treat cancer patients with comorbidities.

The teams most recent findings add to a robust legacy of studies utilizing the ICSBCS biorepository that are clarifying the role of genetic ancestry related to breast cancer risk, added co-author Dr. Lisa Newman, chief of the Section of Breast Surgery at Weill Cornell Medicine and NewYork-Presbyterian/Weill Cornell Medical Center, professor of surgery at Weill Cornell Medicine and ICSBCS medical director and founder.

The investigators are now looking more deeply at gene expression differences to determine the master regulators of the pathways they identified and performing single cell analysis to learn more about the tumor microenvironment. We want to get to the bottom of the molecular features driving disparities in TNBC before we move our work into the clinical space, Dr. Davis said.

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Biological Links Identified Between an Aggressive Breast Cancer Type and African Ancestry - Weill Cornell Medicine Newsroom

Members Bradley Bernstein, M.D., Ph.D., Paula Cannon, Ph.D., Howard Chang, M.D., Ph.D., and Ahmad (Mo) Khalil, Ph.D., will guide advancement of the company's epigenetic editing platform and key programs

Scientific Advisors join Chroma Founders, Luke Gilbert, Ph.D., Keith Joung M.D., Ph.D., David Liu, Ph.D., Angelo Lombardo, Ph.D., Luigi Naldini, M.D., Ph.D., and Jonathan Weissman, Ph.D., expanding the company's world-class team of leaders in genomic medicine

CAMBRIDGE, Mass., Sept 20, 2022 /PRNewswire/ -- Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced the formation of a Scientific Advisory Board (SAB) comprising renowned leaders in epigenetics, cell and gene therapy, and synthetic biology: Bradley Bernstein, M.D., Ph.D., Paula Cannon, Ph.D., Howard Chang, M.D., Ph.D., and Ahmad (Mo) Khalil, Ph.D. The SAB members will provide key input to Chroma as the company advances its programs addressing a wide range of diseases.

"Each of these distinguished experts will be instrumental as we unlock the potential of epigenetic editing therapeutics," said Catherine Stehman-Breen, M.D., Chief Executive Officer of Chroma Medicine. "We are honored to welcome them to the Chroma team and eager to leverage their expertise as we build the future of genomic medicine."

"The SAB is composed of scientific leaders whose seminal research has significantly advanced the fields of genome editing and cell and gene therapy," said Vic Myer, Ph.D., President and Chief Scientific Officer of Chroma. "They bring a wealth of knowledge and experience to Chroma as we continue to advance our platform with the goal of bringing novel single-dose genomic therapeutics to patients."

Members of the Chroma Scientific Advisory Board include:

About Chroma Medicine

Chroma Medicine is a biotechnology company pioneering a new class of genomic medicines that harness epigenetics, nature's innate mechanism for gene regulation, to deliver single-dose therapeutics for patients with genetically driven diseases. The company was founded by the world's foremost experts in genomic research and is led by a veteran team of industry leaders and scientists with deep experience in genomic medicine, drug discovery, and development. For more information, please visit chromamedicine.com or follow the company on LinkedIn and Twitter.

SOURCE Chroma Medicine

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Chroma Medicine Announces Formation of Scientific Advisory Board of Global Experts in Gene Editing and Cell and Gene Therapy - PR Newswire

Artificial intelligence (AI) and big data are transforming healthcare with high-throughput analyses of complex diseases. Machine learning and sophisticated computational methods can be used to efficiently interpret human genomes and other biomarkers, providing insights for patient treatment and with major applications in diagnostics and preventive care.

A personalised treatment plan may include preventive care for diseases that are at a higher risk of developing, for example increased screening for cancer if a patient possesses the BRCA 1 or BRCA 2 gene mutation. Additionally, AI can generate insights from genetic information, biomarkers, and other physiological data to predict how a patient will respond to different treatment options, which may help avoid adverse reactions, reduce the use of expensive or unnecessary treatments on patients that are unlikely to respond, and ultimately reduce hospitalisation and outpatient costs. For more information, GlobalDatas latest report, Precision and Personalized Medicine Thematic Research, provides insight into the most prevalent uses of personalised medicine, new applications, and the healthcare, macroeconomic, and technology themes driving growth.

Big data and bioinformatics can also offer human-centred data to be used for early drug research in lieu of, or in combination with, conventional methods like cell or animal models. This could help streamline the drug discovery process by reducing the time and money spent on inviable drug candidates, especially for conditions that translate poorly between animal models and humans. For example, laboratory mice have historically been utilised in early phase drug trials but are a poor model for genetic diversity and age-related diseases in humans. So, treatments for neurodegenerative and other age-related conditions could greatly benefit from the inclusion of human genetics in research and development (R&D).

The field of oncology has been the most accepting of personalised medicine, though other areas of medicine could greatly benefit from this medical model. Still, major barriers to commercialisation and access are funding and reimbursement. Stockholders want to invest in therapies that have a large patient pool and payers are hesitant to reimburse patients for novel diagnostic tests and treatments that lack the positive clinical data of traditional one-size-fits-all approaches. However, we could see interest in the sector resurge as increasing market competition and advances in technology rapidly drive down the cost of genetic sequencing. Physiological data is also more comprehensive and accessible than ever due to the recent growth of remote patient monitoring devices and wearable tech from the Covid-19 pandemic.

Furthermore, companies are collaborating to reduce development costs and share patient data for research. Recently, Valo Health Inc., a medical technology company, and Kahn-Sagol-Maccabi (KSM), a research and innovation center, announced they will perform joint studies utilising KSMs Tipa Biobank of more than 800,000 samples and Valos drug discovery and development platform Opal. The Tipa Biobank stores live samples, with plans to continue collecting genetic samples from the same subjects over their respective lifetimes. The collaboration provides an opportunity to utilise the growing patient data sector to capitalise on the race to get AI-designed drugs to market and could give Valo/KSM a competitive edge for developing treatments in oncology and for neurodegenerative diseases. Industry collaborations between big market players may also reassure healthcare payers that personalised technology is worth the investment, improving funding and patient identification for new trials and treatments.

Genetic and physiological data can help paint a clearer picture of overall patient health, and it is expected that the demand for preventive medicine will continue increasing as people live longer and the global elderly population grows. Looking to the future, precision and personalised medicine has the potential to expedite drug discovery, improve disease screening, and predict patient responses to treatment options, leading to improved quality of care and reduced overall healthcare costs.

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Personalised medicine and the advantages of big data and AI-based diagnostics - Medical Device Network

BOSTON, Sept. 22, 2022 (GLOBE NEWSWIRE) -- Decibel Therapeutics DBTX, a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced that Laurence Reid, Ph.D., Chief Executive Officer, will participate in a fireside chat at the Jefferies Cell and Genetic Medicine Summit on Friday, September 30, 2022, at 10:00 a.m. ET in New York, NY.

A live webcast of the fireside chat may be accessed by visiting the Investors section of the Decibel Therapeutics website at https://ir.decibeltx.com. An archived replay of the webcast will be available on the Company's website for approximately 90 days following the fireside chat.

About Decibel Therapeutics

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibel's pipeline, including its lead gene therapy program, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all. For more information about Decibel Therapeutics, please visit http://www.decibeltx.com or follow us on Twitter.

Investor Contact:Julie SeidelStern Investor Relations, Inc.julie.seidel@sternir.com212-362-1200

Media Contact:Chris RaileyTen Bridge CommunicationsChris@tenbridgecommunications.com617-834-0936

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Decibel Therapeutics to Participate in the Jefferies Cell and Genetic Medicine Summit - Decibel Therapeut - Benzinga