Improving viral vectors for hemophilia gene therapies by tricking the immune system

San Raffaele Telethon Institute | May 22, 2019

Several gene therapies in late-stage development for hemophilia use adeno-associated virus (AAV) as a vector to deliver a gene that expresses a functional coagulation factor. One limitation of this approach is that most humans have already naturally developed immunity against AAV, which can render the therapy ineffective.

Spotlight

For centuries, farmers have used tools, from plows to tractors, to turn over,
or till, their soil to remove weeds before planting. While this has been a
common and effective method for generations, it can ultimately lead to soil
erosion and loss of essential soil nutrients. Advances in biotech crops and
crop protection products are allowing farmers worldwide a new approach
through conservation – or no-till – agriculture. By leaving the soil untilled,
farmers build up the nutrients in their fields and preserve topsoil, which
boosts yields and helps our environment

Spotlight

For centuries, farmers have used tools, from plows to tractors, to turn over,
or till, their soil to remove weeds before planting. While this has been a
common and effective method for generations, it can ultimately lead to soil
erosion and loss of essential soil nutrients. Advances in biotech crops and
crop protection products are allowing farmers worldwide a new approach
through conservation – or no-till – agriculture. By leaving the soil untilled,
farmers build up the nutrients in their fields and preserve topsoil, which
boosts yields and helps our environment

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MEDICAL

HTL Announces Its Incubator's Equity Investment In GelMEDIX, An Early-Stage Biotech Aiming At Revolutionizing Ocular And Regenerative Therapies

HTL | March 14, 2022

HTL Biotechnology, a pioneer and world leader in the development and production of innovative pharmaceutical grade biopolymers, today announced its incubator's first equity investment in GelMEDIX Inc., an early-stage biotechnology company committed to developing the next generation of ocular and regenerative therapies. This investment supports continued development of the GelMEDIX platform, which enables the delivery of therapeutics from small molecules to cell and gene therapies. Initial research focuses on ophthalmology with lead programs in vision restoring cornea and retina cell therapies and sustained release small molecule therapies. GelMEDIX's most advanced program is focused on developing a sustained release corticosteroid subconjunctival implant which aims to improve patient care in ocular surface inflammation (postoperative pain and inflammation, dry eye disease, allergic conjunctivitis). One drug-loaded implant replaces 70 patient administered eye drops over the course of one month. Instead of using drops, one easy treatment will be administered in the operating theater or in the clinic without any loss of efficacy. In addition to anti-inflammatories, the platform technology can be used for delivery of pro-regenerative therapies that restore ocular health, As such, this product represents one of the most promising innovations deriving from our proprietary hydrogel platform." Reza Dana, M.D., M.P.H., M.Sc., Scientific Co-founder of GelMEDIX. This initial implant constitutes only one of the several research opportunities deriving from GelMEDIX's proprietary photocrosslinkable hydrogel platform, which uniquely enables tunable bioadhesion, tissue regeneration, and biodegradation. HTL's partnership with GelMEDIX also facilitates the development of new applications in regenerative medicine both in ophthalmology and other therapeutic areas. HTL's incubator will support GelMEDIX through a direct investment and industrial and scientific support throughout its development thanks to its expertise in biopolymers and ophthalmology sectors. HTL will also produce methacrylate hyaluronic acid, a key component for tailoring application specific parameters across the GelMEDIX pipeline including viscosity, bioadhesion, and therapeutic release profiles. Additionally, HTL will help GelMEDIX in the industrialization of its hydrogel production. It is an honor to have the support of such a renowned company as HTL. Beyond the financial aspect, its keen understanding of ophthalmology issues, its industrial know-how and the high quality of its products are all crucial assets to accelerate GelMEDIX's development." Arthur Driscoll, President and Chief Development Officer of GelMEDIX. HTL's incubator is a financial vehicle allowing HTL to take minority investments in innovative biotechs in the biopolymer sector, either as seed funds or as series A investments. The incubator also aims to support these biotechs thanks to HTL's unique knowledge and expertise in the production of biopolymers. Innovation is the core of HTL's DNA, which is why we aim at supporting tomorrow's medicine by investing in biotechs that are pushing away the limits of biopolymer use in the medical sector, We are really excited about this partnership with GelMEDIX, which is a perfect example of the type of biotechs to which we wish to provide strategic and financial support". Charles Ruban, Deputy CEO of HTL. This incubator represents one of the strategic axes of HTL's ambitious R&D strategy which positions the French company as the global driver of innovation in the biopolymer sector, developing new markets and applications for biopolymers to address unmet medical needs. The company also relies on its state-of-the-art research facility and numerous partnerships with entities at the forefront of world research to nurture its biopolymer platform for the healthcare industry. About GelMEDIX GelMEDIX Inc. is an early-stage biotechnology company committed to innovating the next generation of ocular and regenerative therapies through its proprietary hydrogel platform. GelMEDIX's programs are based upon its photo crosslinked hydrogels, originally developed by Prof. Nasim Annabi (UCLA) and Prof. Reza Dana (Mass Eye and Ear, Harvard Medical School). These hydrogels uniquely enable bioadhesion, tissue regeneration, tunable mechanics, and therapeutic loading across modalities from small molecules to cell and gene therapies. GelMEDIX is developing drug products for indications across the eye focused on cell-based therapies for vision restoration, intraocular sustained release of small molecules and peptides, and in situ forming bioadhesives. GelMEDIX is backed by Safar Partners and HTL Biotechnology along with leading angel investors and is currently raising a Series-A financing. GelMEDIX is based in Cambridge, MA., USA. About HTL HTL is a leading biotech and industrial player in the development and production of innovative, pharmaceutical-grade biopolymers that are used by leading pharmaceutical and medical device companies to transform the lives of millions of patients in multiple therapeutic areas such as ophthalmology, dermatology, medical aesthetics, rheumatology, and urology.

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ConcertAI Creates COVID-19 Resources for Oncology to Support Healthcare Providers, Biopharmaceutical Innovators, and Biomedical Researchers

ConcertAI | October 08, 2020

ConcertAI has launched a set of COVID-19 resources for oncology to support the needs of healthcare providers, biopharmaceutical innovators, and biomedical researchers as they look to maintain patient access to new and critical treatments and improve outcomes during this pandemic. Abstracted sets of aggregated, de-identified real-world data (RWD) will be derived from CancerLinQ Discovery™– a project of CancerLinQ LLC, a wholly owned nonprofit subsidiary of the American Society of Clinical Oncology (ASCO). The initiative will provide actionable RWD and aligned AI technologies to improve healthcare operations and augment COVID-19 decision-making for oncology care providers. The solutions will also support the ability of critical oncology biopharma innovators to more rapidly redesign stalled clinical research programs and reconsider approaches to new clinical studies based on changes to standard of care, greater use of telehealth tools, and the vulnerabilities of specific groups of cancer patients. Specifically, healthcare providers will gain high-value insights to guide their responses to COVID-19 within their practice settings and biopharma innovators will be able to reinitiate studies and inform the redesign of new studies. ConcertAI will combine CancerLinQ Discovery clinical data with administrative payer claims data to create a complete longitudinal view of a focused population of cancer patients under active treatment.

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RESEARCH

PRISM BioLab, enters multi-project drug discovery collaboration with Roche and Genentech

PRISM BioLab Co., Ltd. | January 05, 2022

PRISM BioLab, a Japan based biotechnology company with a proprietary peptide mimetic small molecule drug discovery platform, announced it has entered into a multi-target research collaboration and licensing agreement with Roche and Genentech, a member of the Roche Group. Under the terms of the agreement, PRISM BioLab will provide its proprietary library of peptide mimetic small molecules, the PepMetics Library, for screening against targets selected by Roche and Genentech. Upon identification of hit compounds, Roche and Genentech may elect to further develop and commercialize the compounds. PRISM BioLab is eligible to receive an upfront payment, success-based milestone payments and royalties on future net sales. Specific financial terms are not disclosed. About PRISM BioLab PRISM BioLab Co., Ltd., is a biotechnology company with a proprietary small molecule drug discovery platform "PepMetics™ Technology". The PepMetics™ molecules are designed to mimic α-helix or β-turn peptides using a unique stable scaffold with corresponding dihedral angles. These motifs are essential for protein-protein interactions within the cell, especially related to transcription and translation. Using this small molecule technology, two clinical-stage assets for cancer and fibrosis have been developed and licensed. Further, PRISM BioLab is working on new drug targets in collaboration with Global and Japanese pharmaceutical companies.

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