Vaccines for Alzheimer’s: exploiting similarities with Down syndrome

Pharmaceutical Technology | September 20, 2019

Vaccines for Alzheimer’s: exploiting similarities with Down syndrome
One approach to overcome continuing challenges facing drug research and development for Alzheimer’s is a vaccine. Swiss AC Immune has developed an Abeta-focused vaccine called ACI-24, which shown to be both effective and safe in pre-clinical models. As well as studying ACI-24 in Alzheimer’s patients, the company is investigating the candidate in Down syndrome patients who have a genetic predisposition to developing Alzheimer’s and are a more homogenous patient group. Believed to be caused by the build-up of plaques in the brain consisting of fibrillary amyloid beta (ABeta) peptides and neurofibrillary tangles of tau proteins, Alzheimer’s disease affects approximately 50 million people globally and is the leading cause of dementia. Despite its prevalence, academic researchers and the pharmaceutical industry have struggled to develop effective therapies against Alzheimer’s disease, with many drugs failing in late stage clinical trials. Research carried out between 2002 and 2012 concluded that the condition has a 99.6% trial failure rate.

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BlockRx is a series of initiatives presented by iSolve, a privately owned Blockchain solution provider for the Life Sciences Industry. The mission of BlockRx is to interconnect systems that do not readily communicate, establish data provenance that satisfies regulatory and business requirements, and create a network of trading partners that are incentivized to facilitate the transfer of information within a secured environment.

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Bayer and Mammoth Biosciences to Collaborate on Novel Gene Editing Technology

Bayer and Mammoth Biosciences | January 10, 2022

Bayer AG and Mammoth Biosciences, Inc., which is harnessing the diversity of nature to power the next-generation CRISPR products, announced a strategic collaboration and option agreement for the use of Mammoth’s CRISPR systems to develop in vivo gene-editing therapies. Mammoth Biosciences’ groundbreaking gene-editing technology is a key enabling technology, as well as a stand-alone therapeutic modality. It will significantly enhance Bayer’s efforts to develop transformative therapies for patients faster and strengthen the company’s recently established new cell and gene therapy platform. Under the terms of the agreement the two companies will start their collaboration with a focus on liver-targeted diseases. “Bringing together Mammoth’s novel CRISPR systems with our existing gene augmentation and our induced pluripotent stem cell (iPSC) platforms will allow us to unleash the full potential of our cell and gene therapy strategy. Partnering with Mammoth’s cutting edge scientific team is a fundamental pillar for our company to improve the lives of patients suffering from conditions that are currently still difficult to treat.” Stefan Oelrich, Member of the Board of Management, Bayer AG and President of the Bayer’s Pharmaceuticals Division “We’re excited to be working together with Bayer, building on the technology leap of our novel CRISPR systems, along with Bayer’s expertise in successful drug development,” said Dr. Peter Nell, Chief Business Officer and Head of Therapeutic Strategy at Mammoth. “This joint effort has the potential to benefit patients by developing CRISPR-based approaches for the clinic with the appropriate urgency, while ensuring scientific excellence and safety.” Cell and gene therapies are the next step in the evolution of drug development. By addressing the root cause of diseases, they are potentially capable of permanently reversing diseases with a one-time treatment. Gene editing serves as a key enabler for cell therapies when used outside the living body and allows therapeutic targeting of a wide range of genetic diseases with a high unmet medical need when used inside the living body. Mammoth Biosciences’ proprietary toolkit of ultra-small Cas enzymes, including Cas14 and Casɸ, allows for expanded high-fidelity gene editing to be combined with targeted systemic delivery. Under the agreement, Bayer gains access to this novel gene-editing technology, which offers the potential of an advanced in vivo applicability due to the ultra-compact size of these novel CRISPR systems. Under the terms of the agreement, Mammoth Biosciences will receive an upfront payment of USD 40 million and is eligible to receive target option exercise fees as well as potential future payments in the magnitude of more than one billion USD upon successful achievement of certain research, development, and commercial milestones across five preselected in vivo indications with a first focus on liver-targeted diseases. In addition, Bayer will pay research funding and tiered royalties up to low double-digit percentage of net sales. The companies are also exploring work on ex vivo projects on a nonexclusive basis. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to drive sustainable development and generate a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2020, the Group employed around 100,000 people and had sales of 41.4 billion euros. R&D expenses before special items amounted to 4.9 billion euros. About Mammoth Biosciences Mammoth Biosciences is harnessing the diversity of life to power the next generation of CRISPR products. Through the discovery and engineering of novel CRISPR systems, the company is enabling the full potential of its platform to read and write the code of life. Mammoth aims to develop permanent genetic cures through best-in-class in vivo and ex vivo therapies and to democratize disease detection with on-demand diagnostics. By leveraging its internal research and development and exclusive licensing to Cas12, Cas13, Cas14, and Casɸ, Mammoth can provide enhanced diagnostics and genome editing for life science research, healthcare, agriculture, biodefense and more. Based in the San Francisco Bay Area, Mammoth Biosciences is co-founded by CRISPR pioneer and Nobel Laureate Jennifer Doudna and Trevor Martin, Janice Chen, and Lucas Harrington. The firm is backed by top institutional investors including Redmile Group, Foresite Capital, Senator Investment Group, Sixth Street, Decheng, Mayfield, NFX, and 8VC.

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Argenx lights up bridges, teases documentary in myasthenia gravis awareness push

Fiercepharma | June 08, 2020

Argenx is launching its first awareness efforts for autoimmune disease myasthenia gravis. The campaign began recently with a virtual conference, light-up nights around the country, a patient website debut and the promise of an upcoming documentary. The kickoff event on June 1, which was also the first day of MG Awareness Month, was a virtual meeting of physician and patient speakers along with the first MG Illuminate night that lit up the Zakim Bridge in Boston in the color teal. Throughout the month, more cities across the country⁠—including Kansas City, Los Angeles, Detroit, Minneapolis and Atlanta⁠—will light up buildings, bridges and other structures in teal to draw attention to the disease. Argenx also debuted its first patient-focused website, MG United, which includes financial and emotional well-being resources along with features such as symptom tracking and career management. The site was built on information gathered through meetings and focus groups with MG patients and caregivers, said Keith Woods, Argenx’s chief operating officer.

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FDA Grants Breakthrough Device Designation to JelikaLite

JelikaLite Corp | January 07, 2022

JelikaLite Corp, a medical technology company, announced that the U.S. Food and Drug Administration has granted the Breakthrough Device Designation to its Cognilum System for the reduction of symptoms of moderate to severe autism spectrum disorder in pediatric patients who are 2 to 6 years of age. The FDA’s Breakthrough Device Program recognizes medical devices that meet the criteria and hold the potential for more effective treatment or diagnosis of life-threatening or irreversibly debilitating human disease or condition. Data from a pilot study suggests that Cognilum, a wearable medical device, can significantly improve the symptoms of children living with autism. If approved, it will offer families access to a home-based, easy-to-use, and cost-effective treatment for children with autism not available anywhere else. Autism is a serious developmental disorder that impacts families around the globe. JelikaLite Corp. believes that it can provide relief to patients and families by reducing children's autism symptoms. Children will be able to better integrate into the society and have more fulfilling lives. Their caregivers can lead less stressful lives and both insurance companies and the government will be able to spend less money on effective intervention, special education and lifelong residential care. The Breakthrough Device Designation for Cognilum was based on the results of a double-blind, placebo controlled clinical trial of 30 pediatric patients with moderate to severe autism. The trial showed both safety and efficacy of this technology. By combining non-invasive brain stimulation with near-infrared light, EEG sensors, and AI personalization platform, Cognilum demonstrated improvements in autism symptoms through gains in communication and daily living skills. "We are thrilled that the FDA recognizes that we are developing a novel technology with a potential to change the landscape in the current approach to Autism. For some of the participants in our active condition, the positive change was so dramatic that their parents expressed their desire to continue the treatment even after the study was over. We recognize that Autism is a spectrum, and no two children are alike; our ultimate goal is to develop a personalized approach to every child based on their unique needs and challenges.” Dr. Eugenia Steingold, Chief Science Officer of JelikaLite JelikaLite’s vision is to bring Cognilum into the homes of families raising children with autism around the globe. This device is truly unique as it seeks to offer a personalized treatment for each child with an integrated feedback loop for both caregivers and doctors. About JelikaLite Based in New York, JelikaLite was launched in 2019 to develop Cognilum™, an innovative solution to permanently reduce children's autism symptoms, enabling better integration into society and reducing lifelong costs. Cognilum™ is a data-device integrated system, where a wearable non-invasive therapeutic medical device that provides neurostimulation treatment is combined with an AI personalization platform. JelikaLite has been founded and run by a multidisciplinary team, with experts in autism clinical therapy, transcranial photobiomodulation, commercialization of medical devices, artificial intelligence, product management, and finance.

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BlockRx is a series of initiatives presented by iSolve, a privately owned Blockchain solution provider for the Life Sciences Industry. The mission of BlockRx is to interconnect systems that do not readily communicate, establish data provenance that satisfies regulatory and business requirements, and create a network of trading partners that are incentivized to facilitate the transfer of information within a secured environment.