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Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha GeneTherapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
The European Commission (EC) has granted conditional marketing authorisation (CMA) for BioMarin Pharmaceutical ’s genetherapy, Roctavian (valoctocogene roxaparvovec), to treat adults with severe haemophilia A (congenital Factor VIII deficiency). Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia genetherapies. Known by the brand name Roctavian, BioMarin’s haemophilia A therapy valoctocogene roxaparvovec could be fairly priced in the range of $1.95–1.96
The US Food and Drug Administration (FDA) has granted Tenaya Therapeutics’ genetherapy TN-201 a fast track designation for the treatment of myosin binding protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). If successful, TN-201 would be a first-in-class disease-modifying genetherapy for MYBPC3-associated HCM.
Working with University of Pennsylvania researcher Jim Wilson, Regeneron aims to develop a nasal spray version of the coronavirus treatment recently cleared by the Food and Drug Administration.
BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia genetherapy – and will now have to see if that can convert that into a viable business in Europe. For now, the list price hasn’t been set and will depend on discussions with reimbursement authorities, according to the biotech.
The EMA’s human medicines advisory committee has recommended that BioMarin Pharmaceutical’s haemophilia A genetherapy Roctavian be approved in the EU, removing one of the last barriers to launch. The post BioMarin genetherapy for haemophilia on course for EU approval appeared first on.
AbbVie has expanded its ophthalmology pipeline via a licensing deal with Regenxbio, paying $370 million upfront to get its hands on a genetherapy for wet age-related macular degeneration (AMD) and other eye diseases. The post AbbVie spies potential in Regenxbio eye disease genetherapy appeared first on.
Shares in VBL Therapeutics have lost around 78% of their value in pre-market trading after the company reported a phase 3 trial of its lead genetherapy VB-111 for ovarian cancer failed a pivotal trial. The post VBL craters as ovarian cancer genetherapy fails phase 3 test appeared first on.
Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based genetherapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
Researchers will now be able to speed up the creation of tailor-made libraries that can help in identifying and developing new cell therapies. Both libraries feature up to 10,000 gene fragment combinations, and allow the high-throughput screening and characterisation of new and known sequence variants to develop therapeutic solutions.
It’s been a big week for cell and genetherapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).
Hansa Biopharma and Genethon have entered a research and development collaboration to test imlifidase as a genetherapy treatment in patients with pre-existing neutralizing antibodies.
The hemophilia A genetherapy Roctavian delivers a functional gene to enable the body to make an essential blood clotting protein on its own. Last week, the first genetherapy for severe hemophilia A , Roctavian (valoctocogene roxaparvovec), developed by BioMarin Pharmaceutical Inc.,
The STAb therapy is based on a special type of bispecific antibody secretion. This antibody can detect targets on the tumour cell and the T cell. An artificial bridge created by these antibodies gets therapeutic T cells and tumour cells into contact. By Cytiva Thematic.
“Evidence is now available that most of the US population five years of age and older have antibodies to SARS-CoV-2, the virus that causes Covid-19, either from vaccination or infection, that can serve as a foundation for the protection provided by the bivalent vaccines.”
According to the positive interim findings from the Phase II/III KidCOVE clinical trial, a two-dose primary series of mRNA-1273 offered a strong neutralising antibody response in the young children with a favourable safety profile. Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
The 2 nd GeneTherapy Immunogenicity Summit is the industry’s definitive forum enabling you to better modulate, measure and predict immune response to your genetherapy candidate. The impact of innate and anti-transgene immunogenicity on AAV vector-mediated gene transfer with Genethon.
These subjects received a minimum of two lines of systemic therapy previously, including the combination of an anti-CD20 monoclonal antibody and an alkylating agent. Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva. FL is a kind of indolent non-Hodgkin lymphoma (iNHL).
In a field dominated by antibodies and small molecules, two cell-therapy based approaches have come under the spotlight for showing early signs of efficacy in treating lupus. Apart from the monoclonal antibodies and small molecules, researchers have long held an interest in exploring cell therapies for autoimmune conditions.
Cure Rare Disease’s Rich Horgan discusses the importance of preclinical NAbs screening and helping rare disease patients better understand their eligibility for genetherapies. Neutralising antibodies (NAbs) present a unique challenge to researchers looking to treat patients with AAV treatment.
In baseline seronegative subjects, the updated vaccine offered a superior neutralising antibody response against Omicron (BA.1) Irrespective of previous infection or age, the vaccine-induced potent neutralising antibody responses against the BA.4 Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
Bioassay – A Prerequisite for the Cell and GeneTherapy Development. Over the past few years, investigational new drug (IND) filings for cell and genetherapy product have significantly increased. Companies Offering Bioassay Services for Cell and GeneTherapies.
Voyager Therapeutics has signed another big pharma partner for its TRACER adeno-associated virus (AAV) vector technology, designed to improve the safety of genetherapies, with Novartis taking an option on three neurological disease programmes. billion including fees and milestones.
UCB has made a new foray into the genetherapy space, buying fellow Belgium-based company Handl to get control of its adeno-associated virus (AAV) capsid delivery platform and two research programmes in neurodegenerative diseases. The post UCB revs up its genetherapy drive with Handl acquisition appeared first on.
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Wilson, Penn ink Regeneron pact to use genetherapy tech to deliver COVID-19 antibodies.Wilson, Penn ink Regeneron pact to use genetherapy tech to deliver COVID-19 antibodies … Continue reading →
Clinical-stage biotechnology company Acepodia has raised $100m in a Series D financing round to advance the first-in-class cell therapies being developed using the antibody-cell conjugation (ACC) platform. Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
Merck has agreed to pay $175 million in cash to Kelun-Biotech, a division of the Chinese pharmaceutical firm Sichuan Kelun Pharmaceutical, for the rights to seven preclinical antibody-drug conjugates, a type of cancer drug that has seen some big successes recently. billion.
Al Sandrock’s shock departure from Biogen in November sparked speculation about where he would show up next, and the answer came in today – genetherapy specialist Voyager Therapeutics. The setbacks effectively sent Voyager back to being a preclinical-stage company however, setting it back years from bringing therapies to market.
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of Roche ’s Columvi (glofitamab) to treat relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) in adult patients after receiving two or more lines of systemic therapy.
FDA Puts uniQure’s Hemophilia B GeneTherapy on Leash. In a recent, uniQure faced backlash after its hemophilia B genetherapy got hit with clinical hold by the USFDA. . The Hemophilia B market has several therapies, however, no therapy has been able to kill the disease from its root.
The New York biotech said today that the SIERRA trial of Iomab-B – an antibody armed with a radioisotope designed to deliver targeted treatment to tumours and spare healthy tissues – met its primary objective. However, current regimens based on intensive chemo- and radiotherapy are very aggressive so are not suitable for frail patients.
AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.
Abecma will be used to treat indicated adult patients who have previously received an immunomodulatory agent, an anti-CD38 monoclonal antibody and a proteasome inhibitor. Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
Shape Therapeutics inks genetherapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of genetherapies for Alzheimer’s and Parkinson’s disease.
The liquid portion of the blood (serum or plasma) containing antibodies is separated and tested using the Liason Biotrin parvovirus B19 IgG plus test on a Liason XL analyzer. If parvovirus B19 antibodies are present, they bind to the chemicals and particular particles in the test, producing a light signal.
Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Avrobio genetherapy eradicates toxic substrate in Fabry patient. Enhancing the response to immuno-oncology in pancreatic cancer. Median overall survival in those patients was 5.8
Switzerland-based company Lonza has boosted its antibody-drug conjugates (ADCs) offering with the acquisition of Dutch biotechnology company Synaffix for a total consideration of $172m (€160m). The deal includes $107.17m (€100m) of initial financial consideration in cash and an additional $64.3m (€60m) in performance-based consideration.
As against a 50µg booster dose of mRNA-1273 in priorly uninfected subjects, mRNA-1273.214 demonstrated superior neutralising antibody response against Omicron BA.1 Irrespective of previous infection or age, the vaccine also showed neutralising antibody responses against the BA.4 1 subvariant.
AstraZeneca has inked an oncology deal with Daiichi Sankyo to jointly develop and commercialize an antibody-drug conjugate DS-1062 (trastuzumab deruxtecan), worldwide except in Japan. No relief to Solid Biosciences as FDA demands more data to lift the hold on the company’s DMD genetherapy trial.
billion partnership with US biotech Sutro Biopharma for a series of antibody-drug conjugates for cancer, focusing on applications in patients who don’t respond to current immunotherapies. Astellas has agreed a $1.36 The alliance includes an upfront payment of $90 million, backed by $422.5
Discover the top six key takeaways from JP Morgan's 2024 conference, focusing on dealmaking trends in big tech, life science, AI, and advancements in cell and genetherapies, as well as the future of Antibody-drug conjugates (ADCs). Stay informed with the latest industry insights.
miRecule and Sanofi have entered a strategic partnership and exclusive licence agreement for the development and commercialisation of an antibody-RNA conjugate (ARC) to treat facioscapulohumeral muscular dystrophy (FSHD). Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
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