Sarepta DMD gene therapy could improve patients’ motor function

Pharmaphorum Media Limited | March 25, 2019

Sarepta Therapeutics announced potentially game-changing results from its Duchenne muscular dystrophy gene therapy candidate, with a small phase 1 trial showing an improvement in patients’ motor function – something that has never been seen before in a clinical trial. These results come from a very small trial involving only four patients aged between four and seven, but Sarepta thought they were strong enough to merit a specially convened call with investors and journalists. The efficacy results were also backed by biomarkers suggesting the therapy is working as planned. In 2016 the FDA controversially approved Sarepta’s Exondys 51 (eteplirsen), which works in a completely different way by getting a patient’s body to ignore instructions for the defective protein that causes the devastating disease. At the time of approval, there was a lack of evidence about the drug on efficacy in terms of patients’ ability to walk and move, with the regulator accepting surrogate endpoint data showing an increase in skeletal muscle in some patients instead.

Spotlight

Since its establishment in 2010, the HGSG has brought together many of the UK’s key individuals and organisations in the field of genetic research and its application to medicine. We have worked together to share knowledge on emerging technologies and existing procedures, to identify the practical barriers that need to be overcome for the NHS to reap the significant potential benefits that genomic technologies can bring, and to suggest potential solutions.

Spotlight

Since its establishment in 2010, the HGSG has brought together many of the UK’s key individuals and organisations in the field of genetic research and its application to medicine. We have worked together to share knowledge on emerging technologies and existing procedures, to identify the practical barriers that need to be overcome for the NHS to reap the significant potential benefits that genomic technologies can bring, and to suggest potential solutions.

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RESEARCH

Evelo Biosciences Presents Data on EDP1815 Mechanism of Action and Supporting Ongoing Clinical Development for Inflammatory Diseases

Evelo Biosciences, Inc. | January 18, 2022

Evelo Biosciences, Inc. a clinical stage biotechnology company developing SINTAX™ medicines as a new modality of orally delivered treatments for inflammatory disease, today announced data for EDP1815, the Company’s lead product in inflammation, detailing its mechanism of action and supporting further clinical development in patients with psoriasis and atopic dermatitis. The data were presented in two posters on Saturday, January 15, 2022, at the 2022 Winter Clinical Dermatology Congress in Koloa, Hawaii. “The data presented today build on the substantial evidence accumulated through our clinical and preclinical work, deepening our understanding of the mechanism by which EDP1815 drives its clinical effects. They explain how an oral, gut-restricted SINTAX medicine can interact with the immune system in the gut, leading to systemic inflammation resolution without immunosuppression or concerning side-effects. We are particularly pleased to share these results with the clinical community as we progress EDP1815 into later stages of development for the treatment of psoriasis and atopic dermatitis, two inflammatory diseases that affect millions of people worldwide.” Douglas Maslin, M.Phil, M.B. B.Chir, Dermatology and Pharmacology Physician at Addenbrooke’s Hospital and Immunology Clinical Lead of Evelo Preclinical data from the studies presented at the Winter Clinical Dermatology Congress confirm that EDP1815 modulates systemic inflammation through its initial interaction with innate immune receptors, including TLR2, leading to downstream changes in circulating immune-cell phenotypes. In addition, the data demonstrate that preclinical effects in Th17 models translate into signs of clinical benefit in psoriasis, and that preclinical effects in Th2 models translate into signs of clinical benefit in atopic dermatitis, supporting further clinical development of EDP1815 in these indications. A Phase 2 study of EDP1815 in patients with mild to moderate psoriasis is ongoing, and a Phase 2 study of EDP1815 in patients with mild, moderate, and severe atopic dermatitis is expected to initiate dosing in the first quarter of 2022. About EDP1815 EDP1815 is an investigational oral medicine being developed for the treatment of inflammatory diseases. It is a non-live pharmaceutical preparation of a strain of Prevotella histicola, selected for its potential to provide systemic pharmacological effects after oral administration with gut-restricted distribution. Being non-live, it has not been observed to colonize the gut or modify the microbiome. Preclinically, EDP1815 had anti-inflammatory effects in models that cover multiple pathways of inflammation, Th1, Th2, and Th17. Clinical results from multiple independent cohorts provide evidence supporting EDP1815’s potential to address Th1, Th2 and Th17-mediated inflammation. About Evelo Biosciences Evelo Biosciences is a clinical stage biotechnology company developing orally delivered product candidates that are designed to act on the small intestinal axis, SINTAX™, with systemic therapeutic effects. SINTAX plays a central role in governing the immune, metabolic, and neurological systems. The Company’s first product candidates are pharmaceutical preparations of single strains of microbes selected for their potential to offer defined pharmacological properties. Evelo’s therapies have the potential to be effective, safe, and affordable medicines to improve the lives of people with inflammatory diseases.

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MEDICAL

TeselaGen Biotechnology Announced the Launch of a New Protein Optimization Toolkit for Automated Biotherapeutic Drug Design and Development

TeselaGen Biotechnology | May 21, 2021

TeselaGen Biotechnology today announced the launch of a new protein optimization toolkit for biotherapeutic drug design and development, introducing significant enhancements to the company’s flagship TeselaGen® OS to form designing and developing pharmaceuticals and biotherapeutics faster and fewer expensive. The new capabilities, easily accessible via the cloud-based platform, simplify the planning of highly complex combinatorial protein libraries and support AI models for optimizing new peptides and proteins. New application programming interfaces (APIs) and integration tools have also been extended to further enhance users’ access to the new capabilities. TeselaGen integrates the facility of AI with one end-to-end platform for design, construction, data gathering, and analysis of bioproduct performance, from pharmaceuticals to food and fabrics, significantly accelerating time to plug and reducing costs. The platform’s DESIGN, BUILD, TEST, and find out modules enable researchers to effectively collaborate across an organization's development pipeline to style and build experiments, standardize and share data, and learn and preserve project results by embedding them during a machine learning model. TeselaGen’s DESIGN is an intuitive, user-interface-driven module that permits scientists to style highly complex combinatorial libraries. With this new release, the planning now supports aminoalkanoic acid parts which will be efficiently mapped to DNA. TeselaGen can then automatically generate biology protocols for efficiently synthesizing and assembling the corresponding DNA libraries. TeselaGen’s DISCOVER now supports AI models which will recommend new peptides and proteins supported by the training of supervised and unsupervised learning models. The platform also supports the modeling of unnatural amino acids and multicriteria optimization of proteins. R&D groups can utilize the TeselaGen OS to hurry the invention process. Datasets are uploaded and arranged within the platform and immediately useful for model building within TeselaGen’s DISCOVER module. TeselaGen has demonstrated that it can increase the planning and build speed of biological products and reduce the prices related to research & development by an order of magnitude. Current partnering companies are using the new capabilities for designing antibodies and optimizing their binding affinity, titer, specific productivity, immunogenicity, or other phenotypic variables of interest. Researchers also are looking to TeselaGen for rapidly engineering new vaccines - using methods like virus-like particles (VLPs), DNA, and RNA vaccines - opening the door to attacking rapidly mutating RNA and retroviruses like influenza, HCV, HIV, or coronaviruses. About TeselaGen Biotechnology TeselaGen Biotechnology has developed the primary artificial intelligence-enabled OS for biotechnology, enabling the event and commercialization of high-performance bioproducts – from pharmaceuticals to food to fabrics – faster and easier than ever. TeselaGen® connects biologists, lab technicians, and bioinformaticians so that they will collaboratively design and build experiments, organize and standardize data then test and continually learn from the info. TeselaGen has been deployed by Fortune 50 companies and emerging innovators in biopharmaceuticals, agriculture, and specialty chemicals. the corporate is privately held and based in San Francisco, California.

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INDUSTRIAL IMPACT

Clover Selected for Inclusion on the Hang Seng Composite Index

Clover Biopharmaceuticals | February 25, 2022

Clover Biopharmaceuticals, Ltd. a global clinical-stage biotechnology company developing novel vaccines and biologic therapeutic candidates, today announced that it has been selected for inclusion in the Hang Seng Composite Index as a constituent stock, effective March 7, 2022. Hang Seng Composite Index Hang Seng Small Cap (Investable) Index Hang Seng Healthcare Index Hang Seng Hong Kong-Listed Biotech Index Hang Seng Stock Connect Hong Kong Index Hang Seng Stock Connect Hong Kong MidCap & SmallCap Index Hang Seng Stock Connect Hong Kong SmallCap Index Hang Seng SCHK Mainland China Companies Index Hang Seng SCHK ex-AH Companies Index The Hang Seng Composite Index (“HSCI”) offers a comprehensive Hong Kong market benchmark that covers about 95% of the total market capitalization of companies listed on the Main Board. Inclusion on the HSCI will allow the company’s stock to be eligible for trading on the Hong Kong Stock Connect, a channel for stock trading between investors in Hong Kong and those in Mainland China. “We are pleased to be added as a constituent stock on the Hang Seng Composite Index. Inclusion on the HSCI, a highly regarded index, will help facilitate the broadening of Clover’s shareholder base, increase trading liquidity and raise global awareness, which will be integral as we complete global regulatory filings for our COVID-19 vaccine candidate and advance our portfolio of innovative vaccine and oncology therapies,” Joshua Liang, Chief Executive Officer and Executive Director of Clover Biopharmaceuticals About SCB-2019 (CpG 1018/Alum) SCB-2019 (CpG 1018/Alum), our COVID-19 vaccine candidate, is anticipated to potentially be one of the first protein-based COVID-19 vaccines commercialized globally through the COVAX Facility. Employing the Trimer-Tag™ technology platform, Clover developed the SCB-2019 antigen, a stabilized trimeric form of the S-protein (referred to as S-Trimer™) based on the original strain of the SARS-CoV-2 virus. Clover created its COVID-19 vaccine candidate by combining SCB-2019 with Dynavax’s (Nasdaq: DVAX) CpG 1018 advanced adjuvant and aluminum hydroxide (alum). About Clover Biopharmaceuticals Clover Biopharmaceuticals is a global clinical-stage biotechnology company committed to developing novel vaccines and biologic therapeutic candidates. The Trimer-Tag™ technology platform is a product development platform for the creation of novel vaccines and biologic therapies. Clover leveraged the Trimer-Tag™ technology platform to become a COVID-19 vaccine developer and created SCB-2019 to address the COVID-19 pandemic caused by SARS-CoV-2.

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