PHARMA TECH

Bayer and Mammoth Biosciences to Collaborate on Novel Gene Editing Technology

Bayer and Mammoth Biosciences | January 10, 2022

Bayer and Mammoth
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.

Spotlight

Welcome to Pharmaprojects’ 2023 review of trends in pharmaceutical R&D. For over 30 years now, I’ve been taking an annual look at the evolution of pharma R&D, and in this article, I’llexamine the state of play at the start of 2023.

Spotlight

Welcome to Pharmaprojects’ 2023 review of trends in pharmaceutical R&D. For over 30 years now, I’ve been taking an annual look at the evolution of pharma R&D, and in this article, I’llexamine the state of play at the start of 2023.

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BUSINESS INSIGHTS, PHARMA TECH

AllianceRx Walgreens Pharmacy Launches RightGuide to Help Minimize Treatment Barriers

Prnewswire | April 27, 2023

AllianceRx Walgreens Pharmacy, one of the largest specialty and home delivery pharmacies in the country, is taking another step in developing innovative solutions with its recent launch of RightGuide, a custom digital engagement program that allows pharmaceutical manufacturers to seamlessly deliver educational support to patients living with rare diseases or chronic conditions. "RightGuide helps minimize potential treatment barriers, from training patients to self-administer injections to addressing financial concerns to mitigating medication side effects. It's a great solution for breaking down complex details to help patients better digest the information," says Tracey James, RPh, chief operating officer, at AllianceRx Walgreens Pharmacy. "With RightGuide, we can deliver the right support – directly into the hands of patients – at the right time." AllianceRx Walgreens Pharmacy created RightGuide to help patients and their caregivers as they navigate the unknowns and complexities of a new therapy. Understanding barriers may be encountered at various stages throughout treatment, RightGuide offers three distinct campaigns, including RightConnect – Onboarding and access RightStart – Therapy initiation and acclimation RightTrack – Ongoing engagement throughout the duration of therapy "Our patient satisfaction surveys indicate patients prefer to engage digitally. RightGuide allows us to deliver relevant information to patients exactly when they need it in their treatment journey," says James. "Patients are more likely to retain this information because it is timely, and presented in an understandable, useful format that's easier for patients to follow." James says pharmaceutical partners are excited to receive co-branded, customized content and drug- or condition-specific resources to help facilitate adherence and retention. Pharmaceutical partners also receive campaign-specific reporting to showcase engagement metrics, including how many patients are accessing information and tools. "Personalization drives engagement and engaged patients have stronger outcomes," says James. "RightGuide is another way we're collaborating with our partners to provide intentional, meaningful engagement while optimizing the patient experience and better positioning them for success on therapy." About AllianceRx Walgreens Pharmacy AllianceRx Walgreens Pharmacy provides hope and care for better tomorrows to patients with rare diseases and chronic conditions by delivering medications from its specialty and home delivery pharmacies. Our advanced analytical capabilities, pharmacy expertise and technology solutions enable providers, pharmaceutical manufacturers, and health plans to deliver optimal health outcomes. Through personalized care and clinical programs, along with access to the leading number of limited distribution drugs, patients can get back to what matters most. Headquartered in Orlando, Florida, the company holds several national pharmacy accreditations and has team members across the United States.

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PHARMACY MARKET

Exscientia Highlights "The Future of AI-enabled Drug Discovery" at SLAS Europe

Businesswire | May 23, 2023

Exscientia plc announced two abstracts for oral and poster presentation, as well as the closing keynote to be delivered at the Society for Laboratory Automation and Screening (SLAS) Europe Conference and Exhibition being held May 22-26, 2023, in Brussels, Belgium. These presentations highlight Exscientia’s recent advancements in technology, including the development of computer-aided biology (CAB) approaches, encompassing design of experiments (DoE) and automation for biochemical assay optimisation. The SLAS Conference is Europe’s premiere venue for industry, government and academic researchers, developers and providers of laboratory automation technology innovation. “At Exscientia, our mission is to encode and automate drug discovery. We believe that within this decade the design of all new drug candidates will be augmented with AI. I am delighted to showcase at SLAS Europe how our recent technology advances enable and drive our projects,” said Dr Garry Pairaudeau, Chief Technology Officer at Exscientia. Integrating laboratory automation of synthesis and screening with its AI-driven computational design platform is the logical next step for the company. “This will facilitate the next transformation in speed and productivity, as we aim to bring better medicines to patients faster. We’re looking forward to opening our purpose-built automation studio in Oxfordshire, U.K., later in 2023.” The company’s presentation at SLAS will demonstrate how far along Exscientia is in automating the drug discovery processes. “We believe automation has the potential to radically improve the timely creation of high-quality drug candidates, so that future patients may benefit from innovative science faster,” Pairaudeau stated. Closing Keynote Presentation Title: The future of AI-enabled drug discovery Room/Location: Plenary Theater Date/Time: Thursday, May 25 / 16:30 – 17:30 PM CET This presentation will highlight Exscientia’s distinct approach to molecular design, using an end-to-end patient centric, AI-enabled platform, which has led the way in the precision design of AI-generated molecules resulting in the first AI-designed compounds to enter clinical trials. To reach Exscientia's goal of fully encoding and automating drug discovery, the company is assembling an automated physical laboratory alongside its suite of sophisticated computational design tools, methods and scalable engineering platform. This presentation will highlight Exscientia's current progress in the automation field and the critical steps needed in the implementation of automated, digital laboratories. Abstracts Accepted for Presentation Oral Presentation Title: Computer-aided biology: DoE solutions Track: Frontiers in Technology Room/Location: 231 Date/Time: Thursday, May 25 / 15:30 – 16:00 PM CET Exscientia is building an automated ecosystem of chemistry, biology and compound management, including developing a pipeline of fully automated biochemical assays from assay development onwards. This presentation highlights how CAB has been leveraged to achieve this pipeline, combining DoE (a statistical methodology) with automation to rapidly determine optimum conditions to run assays efficiently through analysis of multi-factorial experimental results. Utilising CAB also allows Exscientia to increase capacity and efficiency across FTEs, time and reagents required to run assays, increase assay robustness and investigate areas of biology not normally studied. Poster Title: Improving assay optimisation and pharmacological sensitivity of a nuclease by employing a computer-aided biology approach Session/Track: Session A; Frontiers in Technology Abstract Number: 1034-A Date/Time: Wednesday, May 24 / 13:30 – 14:30 PM Exscientia researchers demonstrated the practical use of CAB to simultaneously improve an assay’s performance and reduce the reagent requirement, cost and labour for assay development. This presentation highlights the application of CAB to optimise the buffer of a nuclease assay, resulting in a 15-fold enhanced assay signal in low salt buffer conditions, alongside over a three-fold decrease in protein requirement​ and reduced assay development cycle times. The low salt requirement is a previously unforeseen result from CAB use which would have not been predicted from the literature. This highlights the potential for technology to improve assay performance in ways a human alone would not have been able to predict. About Exscientia Exscientia is an AI-driven precision medicine company committed to discovering, designing and developing the best possible drugs in the fastest and most effective manner. Exscientia developed the first-ever functional precision oncology platform to successfully guide treatment selection and improve patient outcomes in a prospective interventional clinical study, as well as to progress AI-designed small molecules into the clinical setting. Our internal pipeline is focused on leveraging our precision medicine platform in oncology, while our partnered pipeline broadens our approach to other therapeutic areas. By pioneering a new approach to medicine creation, we believe the best ideas of science can rapidly become the best medicines for patients.

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BUSINESS INSIGHTS, PHARMA TECH

Specialty Pharma Expert Adopts Name of Global Parent to Reflect Breadth of Offer That Brings a Rare and Special Perspective to the Market

Businesswire | April 26, 2023

Global healthcare and business consulting services provider, Terebellum, has chosen to adopt the name of its global parent, AscellaHealth, to reflect the scale of its operations and breadth of expertise. The business specialises in providing end-to-end solutions to pharmaceutical manufacturers and other industry stakeholders to streamline product commercialisation, improve medication access, reduce costs and enhance patient outcomes for specialty and rare disease patients. Based in Dublin, Ireland and Manchester, UK, former Terebellum provides pre-commercialisation and market access support, supply chain logistics, distribution and fulfilment and unique pharmaceutical financial services to UK and European pharmaceutical manufacturers and healthcare organisations. Adopting the brand name of its parent company, AscellaHealth, will enable the specialty pharma expert to demonstrate its full range of expertise, international insight and distinctiveness of offer. Upon Terebellum’s transition to the AscellaHealth brand identity in its market-facing activities, the company will continue to maintain current business operations, client relationships and dedication to patient-centric care. Craig Caceci, Managing Director at AscellaHealth EU/UK, said: “It’s our strong belief that to optimise outcomes in specialty healthcare a specialised approach is needed, due to the specific challenges associated with rare conditions. “The AscellaHealth name provides an opportunity to highlight how we are perfectly positioned to offer uniquely tailored solutions across the globe to ensure streamlined medication access and the best possible treatment journey for rare and orphan disease patients.” In a recent customer survey carried out by AscellaHealth, 100 percent of respondents agreed that the organisation presents unique value in the market and is uniquely qualified to address their needs. Craig adds: “Supporting patients, life science manufacturers, payers and providers, we bring expertise and care to every requirement to develop and implement novel approaches that ensure patients with rare diseases can get the treatment they need, when they need it most. “Our new brand tagline, ‘a rare and special perspective’, captures our dedication to applying knowledge and expertise to improve health outcomes of rare disease and specialty patients, perfectly.” AscellaHealth, co-founded by Dea Belazi, President & CEO and Bill Oldham, Chairman & CFO in 2013, employs nearly 200 employees worldwide to provide integrated end-to-end solutions that span the entire pharmaceutical lifecycle and beyond, supporting all segments of the specialty pharma industry. Former Terebellum joins US-based pharmacy distribution and patient-management organisation, Optime Care, in adopting the name of parent AscellaHealth, to form one globally-recognised brand for specialty pharma expertise. About AscellaHealth LLC AscellaHealth, a global Healthcare & Specialty Pharmacy solutions organisation, serving patients, life sciences manufacturers, payers, and providers, offers a comprehensive portfolio of uniquely tailored, tech-enabled services supporting complex, chronic conditions or rare diseases that require specialty medications and/or cell and gene therapies. A recipient of numerous industry awards for innovation, and a NASP Strategic Channel Partner of the Year award winner, AscellaHealth’s best-in-class, patient-centric approach is built upon proprietary technology processes for novel programs and services to support the launch of specialty medications and proactively address multiple challenges, optimise clinical health outcomes, and improve quality of life for this patient population. AscellaHealth brings a rare and special perspective to all stakeholders.

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