CELL AND GENE THERAPY

Moderna and Aldevron Announce Expanded Collaboration for mRNA Vaccine and Therapeutic Pipeline

Moderna, Aldevron | May 25, 2021

Moderna, Inc., a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, and Aldevron, LLC, the leading provider of high-quality plasmid DNA, mRNA, and recombinant proteins used for vaccines, gene and cell therapy, gene editing, and diagnostic applications, have announced an expanded collaboration in support of the Moderna COVID-19 Vaccine and additional programs in mRNA therapeutics and vaccines.

Aldevron will specifically provide plasmid DNA to act as the genetic template for the COVID-19 mRNA vaccine and other investigational programs in Moderna's pipeline.

“Aldevron has been a long-standing Moderna partner. We value their cooperation and expertise in the biologics space,” said Juan Andres, Moderna's Chief Technical Operations and Quality Officer. “We are looking to continue our work in this extended partnership.”

“Aldevron's support for the Moderna pipeline spans nearly a decade, and we're incredibly proud of the trust they've got in us,” said Kevin Ballinger, Aldevron's Chief Executive Officer. “Our extensive experience, combined with improved operational efficiencies and recent capacity expansion, puts us in an excellent position to support Moderna's efforts – especially at this critical stage. We look forward to extending our strategic partnership in the future to serve a pipeline of important new programs.”

Aldevron's DNA production continues in its 70,000 square foot GMP facility in Fargo, North Dakota. The design and validation of an additional 189,000 sq ft expansion to Aldevron's 14-acre Breakthrough Campus's GMP facility have been completed, allowing for increased production capability.

About Aldevron
Aldevron is a world-class manufacturing partner in the area of genetic medicine. Michael Chambers and John Ballantyne founded the company in 1998, and it offers essential nucleic acids and proteins used in gene and cell therapies, DNA and RNA vaccines, and gene editing technologies. Aldevron's 600 employees assist thousands of scientists who are working to develop revolutionary treatments for millions of people.

About Moderna
Moderna has evolved from a science research-stage company advancing programs in the field of messenger RNA (mRNA) to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across six modalities, a broad intellectual property portfolio in areas such as mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant. Moderna maintains partnerships with a wide range of domestic & global government and commercial collaborators, allowing for the pursuit of both groundbreaking science and accelerated manufacturing scaling. Recently, Moderna's capabilities came together to allow the authorized use of one of the earliest and most effective COVID-19 vaccines.

Spotlight

Jessica Garcia Fernandez describes her experiences of the UCD MSc Biotechnology programme, including her reasons for taking this course, the modules she studied and her research project.

Spotlight

Jessica Garcia Fernandez describes her experiences of the UCD MSc Biotechnology programme, including her reasons for taking this course, the modules she studied and her research project.

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CELL AND GENE THERAPY

NeuExcell Therapeutics and Spark Therapeutics Announce Research Collaboration Agreement to Develop a Novel Gene Therapy for Huntington's Disease

NeuExcell Therapeutics | September 13, 2021

NeuExcell Therapeutics and Spark Therapeutics, a member of the Roche Group announced a gene therapy collaboration aimed at developing a safe and effective treatment for patients suffering from Huntington's Disease (HD). Under the terms of the agreement, Spark Therapeutics will receive access to NeuExcell's proprietary neuro-regenerative gene therapy platform and capabilities. NeuExcell's research team will collaborate closely with Spark Therapeutics to advance the program. Under the Option License NeuExcell is eligible to receive upfront, license fees, R&D and Sales milestone payments up to approximately $190 million plus product royalties. Under this Agreement, Spark Therapeutics has the option to license the exclusive worldwide rights of the NeuExcell's HD program. The prevailing assumption has been that mammalian adult neurons cannot be replaced, and so therapeutic approaches for brain diseases tend to focus on slowing disease progress. NeuExcell Therapeutics may have unlocked the method for regenerating neural tissue. The company's neuroregenerative gene therapy platform is built around transcription factor-based trans-differentiation technology. The platform seeks to reprogram endogenous glial cells like astrocytes, which surround neurons and are often reactive after neurons are injured or die, into functional new neurons. While neurons cannot divide to regenerate themselves, glial cells are a renewable source for generating new neurons at the site of injury, and at the scale needed to have a meaningful therapeutic impact. NeuExcell is developing adeno-associated viruses (AAVs)-based neuroregenerative gene therapy to regenerate functional new neurons at the site of the neurodegeneration. "At Spark, we understand that in order to break down barriers for people and families affected by genetic diseases, we need to work with like-minded partners that can integrate innovative technologies with our advanced proprietary AAV vector platform," said Joseph La Barge, Chief Business Officer of Spark Therapeutics. "Using our existing expertise in gene therapy development and NeuExcell's neuro-regenerative gene therapy research and capabilities, together we can progress the potential of gene therapy for patients living with Huntington's Disease." Spark Therapeutic's advanced proprietary AAV vector platform targeted to the central nervous system offers the HD research and development (R&D) program a major advantage. At the forefront of gene therapy research for more than two decades, Spark Therapeutics has extensive knowledge and capabilities in this field that it will use to bring the HD program forward. About Huntington's Disease HD is an incurable, hereditary brain disorder caused by a single defective gene on chromosome 4. As the disease affects different parts of the brain, it impacts movement, behavior, and cognition. It becomes harder to walk, think, reason, swallow, and talk. Eventually, the person will need full-time care. The complications associated with HD are usually fatal. About NeuExcell Therapeutics. NeuExcell is a privately held early-stage gene therapy company headquartered in Pennsylvania, USA. Its mission is to improve the lives of patients suffering from neuro-degenerative diseases and CNS injuries. Based upon the scientific work of Prof. Gong Chen (Co-Founder and Chief Scientific Advisor), the Company has developed a potentially disruptive neural repair technology through in vivo astrocyte-to-neuron conversion by introducing neural transcription factors through adeno-associated virus (AAV)-based gene therapy. NeuExcell's pipeline covers major neurodegenerative diseases such as Stroke, Huntington's Disease, Amyotrophic Lateral Sclerosis (ALS), Alzheimer's Disease, Parkinson's Disease, Traumatic Brain Injury, Spinal Cord Injury, and Glioma. About Spark Therapeutics At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases. We currently have four programs in clinical trials. At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease.

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INDUSTRIAL IMPACT

n-Lorem Foundation Partners with Argonaut to Manufacture ASO Medicines for Patients with Nano-rare Diseases

Argonaut Manufacturing Services | February 02, 2022

Argonaut Manufacturing Services, a leading contract manufacturing organization, and n-Lorem, a nonprofit foundation, today announced a new partnership that supports and reduces the cost of manufacturing of n-Lorem’s experimental antisense oligonucleotide medicines developed for nano-rare disease patients for free, for life. “Providing nano-rare patients, who would otherwise be largely ignored by our current drug discovery paradigm, with a personalized approach for free is a remarkable undertaking and one that requires the commitment of many people and organizations. We are pleased to add Argonaut to our growing list of the industry’s best to support the highest-quality drug discovery, development, and manufacturing for our patients. Reducing the cost of manufacturing translates into lower per-patient costs, which is important for our non-profit and important for our patients who depend upon us and our partners. At n-Lorem, we are fortunate that our partners can integrate seamlessly and efficiently with us and together we work toward making a difference for our nano-rare patient today and tomorrow.” Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation “At Argonaut, we are incredibly proud to be working with this distinguished group of experts at the n-Lorem Foundation. We are focused on delivering the best contract manufacturing support to the innovators and creators, in the biopharma industry. Partnering with the n-Lorem Foundation provides us with the unique opportunity to apply our leading aseptic drug product manufacturing technology in support of nano-rare patients,” said Wayne Woodard, Founder and CEO of Argonaut. “I have been impressed with Stan’s vision and leadership with n-Lorem's charitable approach, and the overall progress of the foundation in such a short time. We are committed strategic partners as we understand that there are > 7,000 known rare to nano-rare diseases. Stan and I share a vision that these patient groups will have access to the life improving or lifesaving drugs that need to be developed. As such, we are honored to work with n-Lorem.” “For decades, patient care for rare disease and now nano-rare patients has improved with technological advancement. n-lorem has established the best partners in rare disease research, and we at Argonaut will be the final step in the sterile fill finish of these life-saving therapies. Our world class platform and expertise in drug product manufacturing were designed specifically for small batch drug product manufacturing that is specifically valuable for rare and orphan disease. This expertise and leadership is what we bring together with n-Lorem,” said Eric Blair, Chief Commercial Officer at Argonaut. About Argonaut Manufacturing Services, Inc. Argonaut Manufacturing Services is an FDA registered, FDB approved, ISO 13485:2016 cGMP contract manufacturing organization dedicated to providing custom manufacturing and supply chain solutions for life science, molecular diagnostics, and biopharmaceutical companies. Life sciences and molecular diagnostics services include formulation, filling, lyophilization, and kitting, while biopharmaceutical services feature state-of-the-art automated aseptic fill-finish equipment for sterile injectable drugs. All projects are supported by a senior project management group, full analytical quality control service offering and global shipping logistics. From procurement through distribution, Argonaut provides a wide range of flexible solutions for diverse outsourcing needs. About n-Lorem n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility, and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 100 applications for treatment with more than 50 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics.

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MEDTECH

Patritumab Deruxtecan Granted U.S. FDA Breakthrough Therapy Designation in Patients with Metastatic EGFR-Mutated Non-Small Cell Lung Cancer

Daiichi Sankyo | December 24, 2021

Daiichi Sankyo Company, Limited announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to patritumab deruxtecan, a potential first-in-class HER3 directed antibody drug conjugate, for the treatment of patients with metastatic or locally advanced EGFR-mutated non-small cell lung cancer with disease progression on or after treatment with a third-generation tyrosine kinase inhibitor and platinum-based therapies. Lung cancer is the second most common cancer and the leading cause of cancer-related mortality worldwide, with 80% to 85% classified as NSCLC.1,2,3 While the efficacy of targeted therapy with EGFR TKIs is well-established in the treatment of advanced EGFR-mutated NSCLC, which comprises approximately 30% of patients, the development of a broad range of resistance mechanisms commonly leads to disease progression.4,5,6 After failure of an EGFR TKI, platinum-based chemotherapy has limited efficacy with progression-free survival (PFS) of approximately 4.4 to 6.4 months.7 Subsequent salvage therapies after EGFR TKI and platinum-based chemotherapy have PFS of 2.8 to 3.2 months.8 The U.S. FDA’s BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The new medicine needs to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines. The FDA granted the BTD based on data from the dose escalation portion and two expansion cohorts of a three-cohort phase 1 study of patritumab deruxtecan. Extended follow-up data from the dose escalation portion and dose expansion cohort 1 of the study were recently presented at the 2021 American Society of Clinical Oncology annual meeting and published in Cancer Discovery. This is the first BTD for patritumab deruxtecan and the seventh BTD across Daiichi Sankyo’s oncology portfolio. “The Breakthrough Therapy Designation for patritumab deruxtecan acknowledges the need for new treatment approaches to overcome resistance and improve survival in patients with metastatic TKI-resistant, EGFR-mutated non-small cell lung cancer. We are proud that the FDA has once again recognized our innovative science and technology and we look forward to bringing this potential first-in-class HER3 directed antibody drug conjugate to patients with this specific type of lung cancer as quickly as possible.” Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo About Non-Small Cell Lung Cancer Lung cancer is the second most common cancer and the leading cause of cancer-related mortality worldwide, with 80% to 85% classified as NSCLC.1,2,3 There were an estimated 2.2 million new cases of lung cancer and 1.8 million deaths in 2020.9 NSCLC is diagnosed at an advanced stage in more than 50% of patients and often has a poor prognosis with worsening outcomes after each line of subsequent therapy.10,11,12 The introduction of targeted therapies and checkpoint inhibitors in the past decade has improved the treatment landscape for patients with advanced or metastatic NSCLC. For patients with advanced EGFR-mutated NSCLC, targeted therapy with EGFR TKIs offer higher response rates and PFS compared to chemotherapy.13 However, most patients eventually develop resistance to these therapies and subsequent therapy after EGFR TKI with platinum-based chemotherapy have limited efficacy with PFS of approximately 4.4 to 6.4 months.7,14 Subsequent salvage therapies after EGFR TKI and platinum-based chemotherapy have PFS of 2.8 to 3.2 months.8 New treatment approaches are needed to overcome resistance and improve survival in this subtype of NSCLC. About HER3 HER3 is a member of the EGFR family of receptor tyrosine kinases, which are associated with aberrant cell proliferation and survival.15 Approximately 25% to 30% of lung cancers have an EGFR-activating mutation, and it is estimated that about 83% of all NSCLC tumors express the HER3 protein, which can be associated with an increased incidence of metastases, reduced survival and resistance to standard of care treatment.16,17,18 Currently, no HER3 directed medicines are approved for the treatment of cancer. About the Phase 1 Non-Small Cell Lung Cancer Study The global, multicenter, open label, two-part phase 1 study is evaluating patritumab deruxtecan in previously treated patients with metastatic or unresectable NSCLC. The dose escalation part of the study evaluated patients with EGFR-mutated disease either with progression on osimertinib or T790M-negative after progression on erlotinib, gefitinib or afatinib. The primary objective of this part of the study was to assess the safety and tolerability of patritumab deruxtecan and determine the recommended dose for expansion. The dose expansion part of the study is evaluating patritumab deruxtecan at the RD in three cohorts. Cohort 1 includes patients with locally advanced or metastatic EGFR-mutated NSCLC who experienced disease progression after taking one or more EGFR TKIs and one or more platinum-based chemotherapy regimens. Cohort 2 includes patients with squamous or non-squamous NSCLC without EGFR-activating mutations following platinum-based chemotherapy and following an anti-PD-1 or anti-PD-L1 antibody regimen. Cohort 3 includes patients with NSCLC with EGFR-activating mutations including any histology other than combined small cell and non-small cell lung cancer; patients in Cohort 3 are randomized 1:1 to receive the 5.6 mg/kg RDE regimen or an escalating up-titration regimen of patritumab deruxtecan. The primary objective of the dose expansion part of the study is to assess efficacy of patritumab deruxtecan as measured by confirmed objective response rate assessed by blinded independent central review. Secondary study endpoints include investigator-assessed ORR, safety and pharmacokinetics. The study enrolled patients at multiple sites in Asia, Europe and North America. For more information, visit ClinicalTrials.gov. About Patritumab Deruxtecan Patritumab deruxtecan is one of three lead DXd ADCs in the oncology pipeline of Daiichi Sankyo. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, patritumab deruxtecan is comprised of a fully human anti-HER3 IgG1 monoclonal antibody attached to a topoisomerase I inhibitor payload via a stable tetrapeptide-based cleavable linker. Patritumab deruxtecan is currently being evaluated in a comprehensive development program across multiple cancers as both a monotherapy and in combination with other anticancer treatments. The development program includes HERTHENA-Lung01, a pivotal phase 2 study in patients with locally advanced or metastatic EGFR-mutated NSCLC previously treated with a TKI and platinum-based chemotherapy; a phase 1/2 study in HER3 expressing metastatic breast cancer; a phase 1 study in combination with osimertinib in locally advanced/metastatic EGFR-mutated NSCLC; and, a phase 1 study in previously treated patients with metastatic or unresectable NSCLC. Patritumab deruxtecan is an investigational medicine that has not been approved for any indication in any country. Safety and efficacy have not been established. About Daiichi Sankyo in Oncology The oncology portfolio of Daiichi Sankyo is powered by our team of world-class scientists that push beyond traditional thinking to create transformative medicines for people with cancer. Anchored by our DXd antibody drug conjugate technology, our research engines include biologics, medicinal chemistry, modality and other research laboratories in Japan, and Plexxikon, our small molecule structure-guided R&D center in the U.S. We also work alongside leading academic and business collaborators to further advance the understanding of cancer as Daiichi Sankyo builds towards our ambitious goal of becoming a global leader in oncology by 2025. About Daiichi Sankyo Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose “to contribute to the enrichment of quality of life around the world.” In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an “Innovative Global Healthcare Company Contributing to the Sustainable Development of Society.”

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