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Cyagen and Neurophth Therapeutics have entered a strategic partnership to jointly develop next-generation AAV genetherapy vectors for specific kinds of genetic ophthalmic ailments. Additionally, Neurophth will oversee the clinical trials and marketing of genetherapy products developed leveraging the new AAV capsids of Cyagen.
US-based biotech company Gatehouse Bio is extending its collaboration with AstraZeneca for developingRNA therapeutics to treat heart failure with preserved ejection fraction (HFpEF). It is expected to expedite the development of a wide range of new therapeutics using the small non-coding area of the genome that is not explored.
How and When to Incorporate PK Design into Your GeneTherapyDevelopment Plan. Genetherapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. GeneTherapy Definition.
A new intracellular drug delivery centre will be established in the UK to support potential ribonucleic acid (RNA) vaccines and therapeutics , as well as the development of innovative drug delivery technologies. It will focus on studying and developing new lipid nanoparticle (LNP) formulations for the delivery of RNA medicine.
The deal will see Polyplus join the German life science group’s portfolio allowing the latter to leverage expertise in transfection reagents and plasmid DNA for genetherapy. Polyplus, based in Strasbourg, France, produces key components in the production of viral vectors used in cell and genetherapies.
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 genetherapies. New investors included research and development partner Novo Nordisk, Lee Family Office (Asia) and Woodline.
Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. This alliance is utilising the Axiomer ribonucleic acid (RNA) editing platform of ProQR to address ailments affecting the liver and nervous system. By Cytiva Thematic.
The collaboration is aimed at discovering and developing new advanced treatments for genitourinary (GU) tumours as well as other related serious diseases using ReviR’s RNA-targeted technologies. The company’s VoyageR AI platform integrates computational methods to drug traditionally undruggable RNA targets.
GreenLight Biosciences has entered a partnership with the US National Institutes of Health (NIH) for the development of Covid-19 vaccines, which offer broader protection against new variants and with durable effects. They intend to develop vaccines that provide lasting immune responses compared to existing vaccines.
from Flanders Innovation & Entrepreneurship (VLAIO) to further advance its oncology portfolio targeting RNA. It will also support the firm’s preclinical effort on the long non-coding RNA (LncRNA) programme, FTX-001, that targets MALAT-1. Flamingo Therapeutics has received a research grant of €1.7m
Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. Ultragenyx uses adeno-associated virus 8 (AAV8) genetherapy to induce stable OTC gene expression. Future Possibilities.
Biotechnology company Ochre Bio has raised $30m in a Series A financing round to developRNAtherapies for chronic liver diseases. It also intends to turn the insights from its research into RNA-based drug candidates, and they will be tested in human livers at the company’s recently opened ‘Liver ICUs’ in the US.
Targovax has unveiled plans to rebrand as Circio , reflecting its strategic shift to focus on expediting the development of its innovative circular RNA (circRNA) platform. Initially reported in 2011, CircRNA is a naturally occurring class of RNA. Topic sponsors are not involved in the creation of editorial content.
The amount is intended to support the company's growing portfolio of RNA technologies and accelerate efforts to develop various treatment approaches in the genetherapy field.
Orbital Therapeutics has raised $270m in a Series A round led by ARCH Venture Partners to advance a portfolio of programmable RNA therapeutics. Orbital will use the new funding to increase the application of RNA-based medicines for use in the fields of new vaccines, immunomodulation and protein replacement.
Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.
At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and GeneTherapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Around 40% of clinical holds are for genetherapy programs.
Myeloid Therapeutics has raised $73m to support the continued clinical development of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. The financing will help to fast-track the development of other in vivo programming candidates into clinical trials.
Charles River Laboratories International, Inc and Rznomics Inc, a South Korea-based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, have established a viral vector contract development and manufacturing organization (CDMO) partnership.
The first subject was dosed in the trial, which is designed to evaluate the safety, tolerability and immunogenicity of a nucleoside-modified RNA-based combination vaccine approach. Fast Track status aids in the development and speeds up the review of new therapies and vaccines for treating or preventing serious ailments.
DTx-1252 is a fatty acid ligand conjugated oligonucleotides (FALCON) small interfering RNA (siRNA) therapeutic which represses the PMP22 gene in Schwann cells. We look forward to the continued development of DTx-1252.” Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
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). The anti-DUX4 RNAtherapy was discovered using the DREAmiR platform of miRecule.
GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The discovery alliance, with a preliminary research period of four years, will aid GSK in progressing up to eight programmes and Wave to develop up to three programmes.
in a project agreement from the US government for developing self-amplifying RNA (saRNA) vaccine technology against advanced and emergent viral threats. Development of vaccines to Phase I trials under the five-year $59m prototype project comprises additional $28.4m in milestone payments. By Cytiva Thematic.
Rona Therapeutics has entered a partnership with Keymed Biosciences to discover and develop first-in-class siRNA [a double-stranded RNA molecule that is non-coding] therapeutics for glomerulonephritis, also known as severe kidney disease. Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
4/5 strain messenger RNA (mRNA). 4/5 Omicron strain is being developed in line with the latest recommendations from the Food and Drug Administration (FDA). Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva. The initial vaccine doses of 66 million are anticipated to be supplied this year.
Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The gene-editing technology allows for precise, directed changes to genomic DNA. The system comprises the Cas9 enzyme and a guide RNA.
The RNA platform of Samsung Biologics permitted GreenLight to move from mRNA vaccine conceptualisation to the delivery of released clinical trial material in under two years. The latest development comes after GreenLight signed a partnership with the US National Institutes of Health (NIH) to develop Covid-19 vaccines against new variants.
MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. BioMarin may then license the assets fully to develop and commercialise them across the globe.
These 'updated boosters' comprise the SARS-CoV-2 virus’ two messenger RNA (mRNA) components, one from the initial strain and the other one common between the BA.4 With the latest development, the monovalent mRNA Covid-19 vaccines are no longer indicated for use as a booster for people aged 12 years and above.
The act also created an industry that didn’t exist in the United States before its enactment, enabling the formation of companies to develop and commercialize therapies for rare diseases. That said, a lot has changed in science and drug development since 1983. The human genome was sequenced in 2003.
To build its ophthalmology portfolio, Japan’s Otsuka Pharmaceuticals has teamed with RNA editing biotech Shape Therapeutics to develop adeno-associated virus genetherapies for ocular diseases.
Moderna has received provisional registration from the Australian Therapeutic Goods Administration (TGA) for its messenger RNA (mRNA) Covid-19 vaccine, Spikevax, for kids aged six months to five years. Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva.
Circio, a biotech firm specializing in circular RNA-based genetherapy, has unveiled two posters showcasing in vivo proof-of-concept for its circVec platform at ASGCT 2024.
Solid Biosciences has entered a strategic research collaboration with Phlox Therapeutics to expedite the development of new therapies for rare cardiac diseases. Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM).
The US Food and Drug Administration (FDA) has given orphan drug designation to SiSaf’s siRNA [a double-stranded RNA molecule that is non-coding] therapeutic, SIS-101-ADO, for the treatment of autosomal dominant osteopetrosis type 2 (ADO2), a rare and serious skeletal disorder in children. There are no approved treatments currently available.
Novartis and Voyager Therapeutics have struck a licensing deal that will see Novartis pay Voyager $100 million upfront to developgenetherapy candidates targeting Huntington’s disease (HD) and spinal muscular atrophy (SMA). billion with preclinical, development, regulatory and sales milestones. Sandrock, Jr.,
In hemophilia patients, inhibitors are antibodies that interfere with the effectiveness of clotting factor replacement therapies. Some individuals with hemophilia A may develop inhibitors against factor VIII, while those with hemophilia B can develop inhibitors against factor IX. Among these are three genetherapies.
Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Moderna will also assume further development, manufacturing, and commercialisation responsibilities on exercising a target option.
TriClip G4 System Manufacturer/developer : Abbott Medical Date of FDA approval : April 1, 2024 Approved for : Tricuspid regurgitation (TR). Abbott Spinal Cord Stimulation (SCS) Systems Manufacturer/developer : Abbott Medical Date of FDA approval : May 30, 2024 Approved for : Chronic, hard-to-manage pain in the torso, arms and legs.
In the ever-evolving landscape of pharmaceutical development, the complexity of early phase clinical trials is increasing. This surge in complexity is driven by escalating drug development costs, heightened competition and advanced scientific innovations. And that has a lot of downstream consequences.” This trend, which Dr.
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.
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