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January 9th

San Francisco State Biology Professor Michael Goldman Receives Two Awards from California State University (CSU) System—One for Exceptional Faculty Service and One for Superb Leadership

BioQuick News has just learned that, on January 5, 2019, one of the publication’s Science & Medicine Advisors, San Francisco State University (SFSU) Professor of Biology and Former SFSU Dean of Biology Michael Goldman (see photo here and below), PhD, was recognized with two separate awards for his exemplary contributions to biotechnology programs in the California State University (CSU) system. Dr. Goldman was the sole recipient of the 2019 Andreoli Faculty Service Award, and he was also only the third recipient in the program’s long history of the CSUPERB Leadership Award, granted to him “in recognition of his dedication to and steady leadership of the CSUPERB community, ushering in an era of growth for the organization,” the selection committee wrote. CSUPERB is the CSU Program for Education & Research in Biotechnology (https://www2.calstate.edu/impact-of-the-csu/research/csuperb). Dr. Goldman has a long history with CSUPERB. Chosen to represent SFSU in the program’s Faculty Consensus Group in 2003, he was elected to the group’s Strategic Planning Council two years later and in 2010 became the chair, a position he held for eight years. He stepped down as chair in 2018, but that hasn’t stopped him from continuing to serve on the Strategic Planning Council. Asked what accomplishments he’s most proud of during his years leading the group, Dr. Goldman has a distinctly student-oriented focus. “We’ve been able to be pretty influential in pushing new ways of learning science such as active learning,” he said, as well as pushing an interdisciplinary focus by broadening representation in CSUPERB beyond biology and biochemistry departments.

January 8th

Relaxin Hormone Reverses Maladaptive Remodeling of Aged Heart by Activating Canonical Wnt Signaling; Discovery May Point to Ways of Treating or Preventing Cardiovascular Disease

As a healthy heart ages, it becomes more susceptible to cardiovascular diseases. Though researchers have discovered that relaxin, an insulin-like hormone, suppresses atrial fibrillation (AF), inflammation, and fibrosis in aged rats, the underlying mechanisms of these benefits are still unknown. In an open-access Scientific Reports article published online on December 6, 2019,, University of Pittsburgh (Pitt) graduate student Brian Martin discusses how relaxin interacts with the body's signaling processes to produce a fundamental mechanism that may have great therapeutic potential. The study, titled "Relaxin Reverses Maladaptive Remodeling of the Aged Heart Through Wnt-Signaling" was led by Guy Salama, PhD, Professor of Medicine at Pitt, and Martin, a graduate student researcher from Swanson School of Engineering's Department of Bioengineering at Pitt. "Relaxin is a reproductive hormone discovered in the early 20th century that has been shown to suppress cardiovascular disease symptoms," said Martin. "In this paper, we show that relaxin treatment reverses electrical remodeling in animal models by activating canonical Wnt signaling, a discovery that reveals a fundamental underlying mechanism behind relaxin's benefits." A better understanding of how relaxin interacts with the body may improve its efficacy as a therapy to treat cardiovascular disease in humans. As the U.S. population ages, the rates of these age-associated diseases are expected to rise, requiring better treatment for this leading cause of death. According to a report (https://healthmetrics.heart.org/wp-content/uploads/2017/10/Cardiovascula... ) from the American Heart Association, the total direct medical costs of cardiovascular disease are projected to increase to $749 billion in 2035.

January 7th

Study of 200,000 Vets Identifies Six Gene Variants Linked to Anxiety and One Is Found Only in African Americans; Study Highlights Advantages of Scale & Diversity in Association Studies; Evidence Also Found for Frequent Co-Morbidity of Anxiety & Depression

A massive genome-wide analysis of approximately 200,000 military veterans has identified six genetic variants linked to anxiety, researchers from Yale and colleagues at other institutions report in an article published online on January 7, 2020, in the American Journal of Psychiatry (https://ajp.psychiatryonline.org/doi/10.1176/appi.ajp.2019.19030256). The article is titled “Reproducible Genetic Risk Loci for Anxiety: Results from ∼200,000 Participants in the Million Veteran Program.” Some of the variants associated with anxiety had previously been implicated as risk factors for bipolar disorder, post-traumatic stress disorder (PTSD), and schizophrenia. The new study further contributes the first convincing molecular explanation for why anxiety and depression often coexist. "This is the richest set of results for the genetic basis of anxiety to date," said co-lead author Joel Gelernter, MD, the Foundations Fund Professor of Psychiatry, Professor of Genetics and of Neuroscience at Yale. "There has been no explanation for the comorbidity of anxiety and depression and other mental health disorders, but here we have found specific, shared genetic risks." Finding the genetic underpinnings of mental health disorders is the primary goal of the Million Veteran Program (MVP), a compilation of health and genetic data on U.S. military veterans run by the U.S. Veterans Administration. The research team analyzed the program's data and zeroed in on six variants linked to anxiety. Five were found in European Americans and one was found only in African Americans. The findings for the African American participants are especially important, says Dr. Dan Levey, PhD, of the VA Connecticut Healthcare Center and Yale University, and a co-lead author of the study.

ERS Genomics Announces Agreement with New England Biolabs to Commercialize CRISPR Gene Editing Tools and Reagents; License Expands New England Biolabs’ Rights to Use and Sell CRISPR/Cas9 Reagents and Tools

On January 7, 2019, ERS Genomics Limited (Dublin, Ireland), which was formed to provide broad access to the foundational CRISPR/Cas9 intellectual property (IP) co-owned by Dr. Emmanuelle Charpentier, announced it has signed an agreement with New England Biolabs (NEB®) (Ipswich, Massachusetts), a global leader in the discovery and production of enzymes for molecular biology applications, granting NEB rights to sell CRISPR/Cas9 tools and reagents. NEB offers the largest selection of recombinant and native enzymes for genomic research, and continues to expand its product offerings into new areas of research, including genome editing. As a provider of CRISPR/Cas9 reagents and tools, NEB’s license from ERS Genomics expands the Company’s rights to use and sell these technologies. ERS Genomics holds an exclusive worldwide license from Dr. Charpentier to the foundational IP covering CRISPR/Cas9. Companies can obtain licenses to this technology for internal research and commercialization of research tools, kits, reagents and genetically modified cell lines and organisms. “New England Biolabs is a recognized world leader in the discovery and production of enzymes for molecular biology applications,“ stated Eric Rhodes, CEO of ERS Genomics. “Adding CRISPR/Cas9 to the company’s portfolio ensures it can continue to support its customers in the latest areas of technology advancement.

January 6th

European Commission Approves Roche’s Kadcyla for the Adjuvant Treatment of People with HER2-Positive Early Breast Cancer with Residual Invasive Disease After Neoadjuvant Treatment

On December 19, 2019, Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the European Commission has approved Kadcyla® (trastuzumab emtansine) for the adjuvant (after surgery) treatment of adult patients with HER2-positive early breast cancer (eBC) who have residual invasive disease in the breast and/or lymph nodes after neoadjuvant (before surgery) taxane-based and HER2-targeted therapy. “Optimal treatment is vital for every patient with early-stage breast cancer, a setting where cures are possible,” said Levi Garraway, MD, PhD, Roche’s Chief Medical Officer and Head of Global Product Development. “This approval of Kadcyla will allow many more women with HER2-positive early breast cancer to be given a transformative treatment that may cut the risk of their disease returning or progressing." The goal of neoadjuvant treatment is to shrink tumors in order to help improve surgical outcomes. Adjuvant treatment aims to eliminate any remaining cancer cells in the body to help reduce the risk of the cancer returning. People who have residual disease after neoadjuvant treatment have a worse prognosis than those with no detectable disease. The approval of Kadcyla in Europe is based on results from the phase III KATHERINE study, which showed that Kadcyla significantly reduced the risk of invasive breast cancer recurrence or death from any cause (invasive disease-free survival; iDFS) by 50% (HR=0.50, 95% CI 0.39-0.64, p<0.001) compared to Herceptin® (trastuzumab) as an adjuvant treatment in people with HER2-positive eBC who have residual invasive disease after neoadjuvant taxane and Herceptin-based treatment. At three years, 88.3% of people treated with Kadcyla did not have their breast cancer return compared to 77.0% treated with Herceptin, an absolute improvement of 11.3%.

January 5th

NIH Researchers Discover Rare Autoinflammatory Disease (CRIA Syndrome) and Determine Its Biological Cause: Mutations in the RIPK1 Gene; Immunosuppressive Drug (Tocilizumab) Is Effective Treatment in Some Cases

Over the last 20 years, three families have been unsuspectingly linked by an unknown illness. Researchers at the National Human Genome Research Institute (NHGRI), part of the National Institutes of Health, and other organizations have now identified the cause of the illness, a new disease called CRIA syndrome. The results were published online on December 11, 2019 in Nature. The article is titled “Mutations That Prevent Caspase Cleavage of RIPK1 Cause Autoinflammatory Disease.” NHGRI Scientific Director Daniel Kastner (photo), MD, PhD, a pioneer in the field of autoinflammatory diseases, and his team discovered CRIA, which has symptoms including fevers, swollen lymph nodes, severe abdominal pain, gastrointestinal problems, headaches, and, in some cases, abnormally enlarged spleen and liver. The disorder has characteristics typical of an autoinflammatory disease, where the immune system appears to be activated without any apparent trigger. Although the condition is not life-threatening, patients have persistent fever and swollen lymph nodes from childhood to old age, as well as other symptoms that can lead to lifelong pain and disability. When confronted by the symptoms of patients, who were first seen at the NIH Clinical Center, researchers looked for infections and cancer as the cause. After those were ruled out, they sought answers in the genome, a person’s complete set of DNA. Dr. Kastner and his team sequenced gene regions across the genome and discovered only one gene — RIPK1 — to be consistently different in all patients. The image here depicts the kinase domain of the RIPK1 protein. Researchers identified a specific type of variation in the patients: a single DNA letter at a specific location incorrectly changed. This change can alter the corresponding amino acid added to the encoded protein.

Wave Life Sciences Reports 12.4% Reduction in Mutant Huntingtin Protein Using Allele-Specific Stereopure Antisense Oligo Targeted at SNP2 in Mutant Huntingtin Transcript; Reports Topline Data from Ongoing Trial & Plans to Initiate Higher-Dose Cohort

On December 30, 2019, Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced topline data from the ongoing Phase 1b/2a PRECISION-HD2 trial evaluating investigational therapy WVE-120102, designed to be the first allele-selective approach to treat Huntington’s disease (HD). In an analysis comparing all patients treated with multiple intrathecal doses of WVE-120102 to placebo, a statistically significant reduction of 12.4% (p<0.05) in mutant huntingtin (mHTT) protein was observed in cerebrospinal fluid (CSF). An analysis to assess a dose response across treatment groups (2, 4, 8, or 16 mg) suggested a statistically significant response in mHTT reduction at the highest doses tested (p=0.03). WVE-120102 was generally safe and well tolerated across all cohorts. These data support the addition of higher dose cohorts, and Wave expects to initiate a 32 mg cohort in January 2020. “This topline analysis has given us the opportunity to evaluate early data from our ongoing dose finding study. The data demonstrate a reduction in mutant HTT and a safety and tolerability profile that supports exploration of higher doses of WVE-120102, with the goal of maximizing mutant HTT reduction and avoiding a negative impact on the healthy huntingtin protein,” said Michael Panzara, MD, MPH, Chief Medical Officer of Wave Life Sciences. “We plan to initiate the 32 mg cohort imminently and look forward to sharing data in the second half of 2020.”

Roche Concludes Acquisition of Spark Therapeutics to Strengthen Presence In Gene Therapy

On December 17, 2019, Roche (SIX: RO, ROG; OTCQX: RHHBY) and Spark Therapeutics, Inc. (NASDAQ: ONCE) (“Spark”) announced the completion of the acquisition following the receipt of regulatory approval from all government authorities required by the merger agreement. Commenting on this important step forward, Severin Schwan, CEO of Roche, said, “We are excited about this important milestone because we believe that together, Roche and Spark will be able to significantly improve the lives of patients through innovative gene therapies. This acquisition supports our long-lasting commitment to bringing transformational therapies and innovative approaches to people around the world with serious diseases.” Spark Therapeutics, based in Philadelphia, Pennsylvania, is a fully integrated, commercial company committed to discovering, developing, and delivering gene therapies for genetic diseases, including blindness, hemophilia, lysosomal storage disorders, and neurodegenerative diseases. Spark Therapeutics will continue to operate as an independent company within the Roche Group. “Today ushers in a new and promising era in the development of genetic medicines for patients and families living with inherited diseases and beyond,” said Jeffrey D. Marrazzo, Co-Founder and CEO of Spark Therapeutics. “Spark and Roche share an ethos of imagining the unimaginable. Together, we have the potential to change the future of medicine and deliver the medicines of tomorrow today. We couldn’t be more thrilled about what’s next.” Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives.

January 4th

Amazing Finding: Eye-Less Red Brittle Star Can See; Only Second Example in All of Biology of Animal Able to See Without Eyes; Deep Red Daylight Pigment of Color-Changing Brittle Star Is Key to Enabling Vision

Scientists have shown for the first time that brittle stars use vision to guide them through vibrant coral reefs, thanks to an unusual color-changing trick. The international team, led by researchers at Oxford University Museum of Natural History, described a new mechanism for vision in the red brittle star Ophiocoma wendtii, a relative to sea stars and sea urchins, which lives in the bright and complex reefs of the Caribbean Sea. The team’s findings were published online on January 2, 2020 in Current Biology. The open-access article is titled “Extraocular Vision in a Brittle Star Is Mediated by Chromatophore Movement in Response to Ambient Light.” This species first captured scientific attention more than 30 years ago thanks to its dramatic change in color between day and night and its strong aversion to light. Recently, researchers demonstrated that O. wendtii was covered in thousands of light-sensitive cells, but the exact behaviors these cells control remained a mystery. The new research shows that O. wendtii is able to see visual stimuli, and that its signature color-change might play an important role in enabling vision. Lauren Sumner-Rooney, PhD, a research fellow at Oxford University Museum of Natural History who studies unusual visual systems, has been working with Ophiocoma for several years at the Smithsonian Tropical Research Institute in Panama and the Museum für Naturkunde in Berlin. Alongside team members from the Museum für Naturkunde, Lund University (Sweden), and the Georgia Institute of Technology (USA), Dr. Sumner-Rooney ran hundreds of behavioral experiments to test the brittle stars' “eyesight.”

Researchers Identify Positions of All Atoms in Clostridium difficile’s Binary Toxin; Results May Serve As Starting Point for Effective Drug Design for Treatment of Often Deadly Bacterial Infection

An open-access article published online on January 2, 2020 in PNAS details a research breakthrough that provides a promising starting point for scientists to create drugs that may cure Clostridium difficile (C. diff) infection-- a virulent health care-associated infection that causes severe diarrhea, nausea, internal bleeding, and potentially death. The article is titled “Structure of the Cell-Binding Component of the Clostridium Difficile Binary Toxin Reveals a Di-Heptamer Macromolecular Assembly.” The C. diff bacteria affects roughly half-a-million Americans and causes nearly 15,000 deaths in the U.S. annually, and results in over $5 billion in health-care-related costs each year in this country. Overuse of antibiotics has increasingly put patients in heath care facilities at risk for acquiring C. diff and made some strains of the bacteria particularly hard to treat. But newly discovered information about a type of toxin released by the most dangerous strains of C. diff is providing researchers with a map for developing drugs that can block the toxin and prevent the bacteria from entering human cells. "The most dangerous strains of C. diff release a binary toxin (image) that first binds to cells and then creates a pore-forming channel that allows the toxin to get inside and do harm," said Amedee de Georges, PhD, the study's principal investigator and a professor with the Advanced Science Research Center at The Graduate Center, CUNY's Structural Biology Iniative. "We were able to combine several increasingly popular biophysical imaging techniques to visualize and characterize every atom of this binary toxin and show us where they are positioned. These details provide a critical and extremely useful starting point for designing drugs that can prevent C.