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Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new geneticmedicines. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The companies entered the initial agreement in September last year. By Cytiva Thematic.
The acquisition marks a significant bet by Lilly on gene therapy's potential to address tough-to-treat neurological disorders like Parkinson's disease.
Biology is opening up new frontiers in medicine. We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics.
Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine. A new frontier in cancer research.
Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of geneticmedicines.
Eli Lilly and Company has signed a definitive agreement for the acquisition of all outstanding shares of precision geneticmedicine firm Akouos for a total deal value of up to nearly $610m or up to $15.50 It is a gene therapy intended to treat hearing loss due to otoferlin gene (OTOF) mutations. for each share in cash.
Facing mounting competition to its top-selling drug Eylea, Regeneron is teaming up with ViGeneron to develop a geneticmedicine for an inherited retinal disease.
The British pharma is deepening its investment in geneticmedicine through a deal for a group of Pfizer’s gene therapy delivery tools, while the New York giant cuts back.
CARsgen Therapeutics has announced a col labor ation with Huadong Medicine to commercialise zevorcabtagene autoleucel (zevor-cel), CT053, in mainland China. It comprises autologous T cells that are modified genetically with a CAR including a complete human anti-BCMA single-chain fragment variant that has a high binding affinity.
Researchers see a greater need for new generation therapeutics to propel precision medicine. The development of innovative therapeutic approaches in healthcare encompass a variety of fields, including gene therapy, personalized medicine, immunotherapy, and advanced biologics.
Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in geneticmedicines.
An 11-year-old boy born deaf can hear after receiving Lilly's geneticmedicine. Others, including biotechs in China and Regeneron in the U.S., are taking aim at the same drug target.
The New York biotech will invest in and develop up to three drugs with Gensaic, an emerging startup aiming to use the viruses that infect bacteria to deliver geneticmedicines.
A deal with biotech startup Capsida, which recently raised $140 million, gives CRISPR access to a Caltech technology that aims to improve the delivery of geneticmedicines.
Lysosomal storage disorders, diseases of cellular metabolism gone wrong, are the focus of a fast-expanding lineup of experimental geneticmedicines, a research boom that recalls biotech's roots.
Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes. Topic sponsors are not involved in the creation of editorial content.
The longtime partners believe that, by combining their technologies, they can create “in vivo” geneticmedicines for nervous system and muscular disorders.
The biotech is the latest in a growing list of geneticmedicine developers to sell themselves near record stock lows amid a challenging funding environment.
Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
Softbank and Fidelity joined a large group of investors in a $525 million Series C investment into Elevate, a high-powered geneticmedicine startup with an unusual business model.
The new facility cost nearly $70 million to build, and is part of a major push by the pharma giant to become a leader in geneticmedicine manufacturing.
Moderna has entered a strategic partnership with Generation Bio for the development of non-viral geneticmedicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral geneticmedicine platform from Generation Bio.
ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.
In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Gene therapy delivery using viral vectors. Sanofi is one of the leading patent filers working with viral vectors.
Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.
Under the deal, Prevail obtains exclusive rights to use Scribe’s CRISPR X-Editing (XE) technologies to develop the medicines. The deal will see the integration of Scribe’s new CRISPR by Design approach and Prevail’s expertise in developing geneticmedicines for neurological disorders for specific genetic targets.
million list price, the highest of any geneticmedicine to come to market. Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25
Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.
Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP. By Cytiva Thematic.
Obesity is a multifactorial disease in which environmental conditions and several genes play an important role in developing this disease. Obesity and genetics. Over a hundred genes and gene variants related to excess weight have been discovered. The rate of new cases of colorectal cancer dropped 23% during this time.
The British drugmaker, which has made geneticmedicine a larger priority of late, added $140 million to a deal to develop up to 10 gene and cell therapies.
Pharmacogenomics (PGx), the study of how genes affect a person’s response to drugs, has brought significant changes to the clinical trial industry. This groundbreaking approach can help tailor medical treatments to an individual’s genetic makeup, considerably enhancing drug efficacy and safety while minimizing adverse drug reactions.
Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers.
The acquisition of Proof Diagnostics, which was formed to develop a COVID-19 test, gives Ginkgo a library of novel enzymes that can be used to make geneticmedicines.
Biotechnology company Hopewell Therapeutics has raised $25m in seed financing to accelerate the development of next-generation lipid nanoparticles for targeted delivery of genomic medicines. Hopewell Therapeutics is engaged in discovering, synthesising and developing advanced ttLNPs to provide next-generation genomic medicines.
A Swedish scientist won the 2022 Nobel Prize in medicine or physiology on Monday for his groundbreaking research into the evolutionary history of humankind. Pääbo unlocked scientists’ understanding of how genes from these extinct relatives have been passed down to present-day humans.
Wilson Bryan, a key decision maker in the agency’s oversight of geneticmedicines, is expected to leave next month amid an organizational revamp of his office, the FDA confirmed.
However, more immeasurable characteristics such as personality, behaviour, and even intelligence are all influenced by genetics to varying degrees. All that DNA is organised into hereditary units called genes, with humans having about 25,000 genes collectively known as the genome. Each human cell has 1.8
The approval allows KALYDECO to be used in infants who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the therapy, on the basis of clinical and/or in vitro assay results. The medicine is currently available for use in more than 30 countries.
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