Syndicate content

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.

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%.

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.

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.

Water Lily Genome Expands Picture of the Early Evolution of Flowering Plants

The newly reported genome sequence of a water lily sheds light on the early evolution of angiosperms, the group of all flowering plants. An international team of researchers, including scientists at Penn State, used high-throughput next-generation sequencing technology to read out the water lily's (Nymphaea colorata) genome and transcriptome (the set of all genes expressed as messenger RNAs). The unusual high quality and depth of coverage of the sequence allowed the researchers to assemble the vast majority of the genome into 14 chromosomes and identify more than 31,000 protein-coding genes. An open-access paper describing the sequence and subsequent analysis was published online on December 18, 2019 in the journal Nature. "Water lilies have been an inspiration to artists like Claude Monet because of their beauty and important to scientists because of their position near the base of the evolutionary tree of all flowering plants," said Hong Ma, PfD, Associate Dean for Research and Innovation, Huck Distinguished Research Professor of Plant Molecular Biology, and Professor of Biology at Penn State, one of the leaders of the research team. "I previously contributed to the sequencing and analysis of the genome of Amborella, which represents the earliest branch to separate from other flowering plants, but Amborella lacks big showy colorful flowers and attractive floral scent, both of which serve to attract pollinators in most groups of flowering plants.

New Photodynamic Therapy Based on Photosensitizers (Photosens and Photodithazine) Induces Immunogenic Cell Death in Mouse Tumor Cells

The world scientific community is waging a difficult and prolonged war on cancer. New research in the field of immunogenic cell death can extend the area of drugs application and ensure patients' protection from relapse after therapy. Cancer treatment is not just the removal of the tumor cells from the body, and chemotherapy. The doctors' aim is to provide a scenario that would prevent tumor cells from proliferating and causing a new disease. For many years, scientists at the Lobachevsky State University of Nizhny Novgorod and the University of Ghent (Belgium) have been engaged in research aimed to minimize the harm to the body after cancer treatment and have been looking for new approaches to treating cancer patients. The project, supported by a grant from the Russian Science Foundation and headed by Dmitry Krys'ko, PhD, leading researcher of the Lobachevsky University's Institute of Biology and Biomedicine, Professor at Ghent University, has yielded its first major results. According to Professor Krys'ko, the existing anti-cancer therapy (chemotherapy, radiation therapy and photodynamic therapy) causes great damage to the body as a whole, while his team's research is aimed at the stimulation of immunogenic cell death, which not only minimizes the damage, but also enhances the efficacy of treatment by involving the body's resources in the fight against cancer. "In this study, we tested some drugs for anticancer therapy based on photodynamic treatment and investigated their new immunogenic properties. We can say that not only the external impact will be used to fight cancer, but also the body itself will engage in the fight by triggering the reactions of the adaptive immune response.

Ratio of Neutrophils to T Cells in Tumor Environment Can Help Predict Which Patients Will Respond to Checkpoint Inhibitor Immunotherapy in Lung Cancer; Study Suggests That Neutrophil-Blocking Drugs May Boost Effectiveness of Checkpoint Inhibitors

For many lung cancer patients, the best treatment options involve checkpoint inhibitors. These drugs unleash a patient's immune system against their disease and can yield dramatic results, even in advanced cancers. But checkpoint inhibitors come with a huge caveat: They only help a small subset of patients. Doctors struggle to predict who these patients are and -- just as important -- who they aren't. Results from a new study published online on December 18, 2019 in JCI Insight could help improve those forecasts. The open-access article is titled “Neutrophil Content Predicts Lymphocyte Depletion and Anti-PD1 Treatment Failure In NSCLC.” After analyzing tumor samples from 28 patients with non-small cell lung cancer(NSCLC), researchers linked a common immune cell with treatment failure. The culprit: neutrophils, the most abundant type of white blood cell. The paper shows that the balance between neutrophils and another type of immune cell -- disease-fighting T cells -- could accurately predict which patients would respond or not. If more neutrophils than T cells were crowded into a tumor, the drugs did not curb the patients' cancers. But if the balance was reversed, checkpoint inhibitors revved up patients' immune systems against their disease. "The study is the first to implicate neutrophils in the failure of checkpoint inhibitors," said senior author Dr. McGarry Houghton, MD, a lung cancer immunologist at Fred Hutchinson Cancer Research Center in Seattle, Eashington. "Our findings also hint at a way to help patients who have this cellular signature." In a mouse model of NSCLC, the researchers administered a drug that decreased the number of neutrophils in and around tumors. That, in turn, boosted the efficacy of checkpoint inhibitors -- T cells now had a clear path to attack diseased cells in the mice.

Syndicate content