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Scribe will receive $40 million upfront from Sanofi in a collaboration initially focused on developing an in vivo gene editing treatment for sickle cell disease.
In this continuing series, BioPharma Dive examines new areas of biotech investment and startup activity, such as next-generation RNA medicines, TYK2 inhibitors and in vivo CAR-T.
CIC biomaGUNE researchers have demonstrated the restoring in vivo of the connection between neurons, yielding functional results Credit: Author: Pedro Ramos / CIC biomaGUNE Research conducted by two groups at the Center for Cooperative Research in Biomaterials CIC biomaGUNE and one at SISSA, Scuola Internazionale Superiore di Studi Avanzati (Italy), (..)
So far, human trial data for gene-editing drugs have been generated for ex vivo therapies, meaning the CRISPR technology is used to modify cells outside the body, which are then reinfused into the patient. — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.
Ginkgo Bioworks (Ginkgo) and the Wisconsin Alumni Research Foundation (WARF) have collaborated to discover next-generation GD2 CAR T-cell therapies to treat solid tumours. Ginkgo will partner with researchers from Wisconsin-Madison to discover new and improved GD2 CAR designs with improved persistence, fitness and proliferation.
Interius BioTherapeutics has received approval from Australia’s Human Research Ethics Committee to begin the first-in-human trial of an investigational in vivo CAR-T therapy designed to treat B-cell malignancies.
Researchers in China have taken the first step towards a new way of treating gastric wounds by using a microrobot combined with the new concept of “in situ in vivo bioprinting” to carry out tissue repair inside the body.
BioMed X has entered a research partnership with Sanofi to leverage artificial intelligence (AI) for drug development. Under the joint research project, a new research team will be set up at the BioMed X Institute in Heidelberg, Germany. The companies will select a varied group of skilled researchers for the project.
Umoja Biopharma has signed a research agreement with IASO Biotherapeutics (IASO Bio) to develop off-the-shelf therapies for haematological malignancies. In the initial stage, the partnership will focus on acute myeloid leukaemia (AML) to boost accessibility to patients.
That was the case when Thilini Gamage was to carry out one of the studies in her doctoral work with Professor Eirik Frengen at the Institute of Clinical Medicine, University of Oslo. They study gene variation and mutations that cause rare genetic diseases. A mutation is a permanent change in the genetic material.
Achieve Safe & Targeted In Vivo Delivery of Payloads with Clarity in Regulations & Appropriate Models to Advance the Next Frontier of In Vivo Editing from Research to Clinic
Last month, the South San Francisco, California-headquartered company presented preclinical data on its KRAS inhibitor pipeline, which includes QTX3034 and QTX3046 at the Annual Meeting of the American Association for Cancer Research in Orlando, Florida. Quanta Therapeutics has raised $50.7 billion in 2029.
In this episode, Vera spoke with Tim Culp, PhD, Vice President of Research and Development at Interius BioTherapeutics, a Philadelphia-based biotech company. Dr. Culp completed a PhD from the Pennsylvania State University, College of Medicine for his research on human papillomaviruses.
An ex-vivo lentiviral vector (LVV) gene therapy, Zynteglo’s every dose is made by genetically modifying the bone marrow stem cells of the patient to produce functional beta-globin. thalassemia in adult and paediatric patients. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
Furthermore, the current understanding of beta cell function is mostly derived from studies of ex vivo isolated islets in rodent […]. However, the highly regulated process of insulin secretion, especially its molecular features and the stimuli behind this process have not yet been fully understood.
A new research paper, titled "In vitro and in vivo effects of zoledronic acid on senescence and senescence-associated secretory phenotype markers," was published on the cover of Aging
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. Under the deal, both companies will partner on the research and preclinical studies, which will be funded by Moderna.
ProPhase Labs’ wholly owned subsidiary ProPhase BioPharma has entered a two-year collaboration agreement with Dana-Farber Cancer Institute for further research and development of Linebacker-1 (LB-1). The partnership deal provides for first and second-year research plans.
EdiGene Biotechnology USA has moved into a new Research & Development Center in Waltham, Mass. to advance its proprietary LEAPER tech into in vivo RNA editing therapies: with an initial focus on ophthalmology and the central nervous system.
Under the collaborative research and development agreement (CRADA), respirable dry powder formulations of HMW-HA will be developed for prevention and treatment. This follows earlier research which demonstrated that HMW-HA yielded dry powder formulations with desirable aerosol performance when formulated with thin film freezing technology.
Scribe Therapeutics has entered a strategic collaboration with Eli Lilly and Company subsidiary Prevail Therapeutics for accelerating in vivo CRISPR-based therapies to target the causes of serious neurological and neuromuscular diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
BioNTech has ended its research collaboration with Matinas after its oral mRNA vaccine failed to demonstrate preclinical activity. The in vivo study, conducted in mice, involved a phosphatidylserine-containing nano-formulation of mRNA supplied by BioNTech, distinct from traditional lipid nanocrystals (LNCs).
Pfizer and Beam Enter Exclusive Multi-Target Research Collaboration to Advance Novel In Vivo Base Editing Programs for a Range of Rare Diseases Pfizer and Beam Enter Exclusive Multi-Target Research Collaboration to Advance Novel In Vivo Base Editing Programs for a … Continue reading →
A research team led by Prof. Gong Chen at Jinan University, Guangzhou, China recently reported the first non-human primate study demonstrating successful in vivo neural regeneration from brain internal glial cells for stroke repair. This work was […].
Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). Other companies – notably Vertex Pharma/CRISPR Therapeutics and bluebird bio – are taking an ex vivo approach to SCD and thalassaemia and are much closer to market.
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Intellia, LogicBio fuel hope for in vivo gene editing with new animal data in rare diseases: report.Intellia, LogicBio fuel hope for in vivo gene editing with new … Continue reading →
Developing both in vivo and ex vivo gene editing medicines, Editas Medicine is primarily involved in the haematology and oncology therapy areas. However, not all innovations are equal and nor do they follow a constant upward trend. Editas Medicine is one of the most important players concerning innovation surrounding CRISPR nucleases.
Roche will also make further payments to Avista during the collaboration’s research phase and clinical, sales-based milestone and royalty payments for resulting products. Avista's computationally steered, in vivo scAAVengr platform uses a high-throughput method with the integrated quantitative validation of new cell-specific AAVs.
Innovation S-curve for artificial Intelligence in the pharmaceutical industry In-silico drug discovery is a key innovation area in artificial Intelligence Drug research starts with the discovery stage that involves in vivo and in vitro models to shortlist lead drug compounds.
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Sanofi pays $160M for mRNA tech to reprogram T cells in vivo.Sanofi pays $160M for mRNA tech to reprogram T cells in vivo ntaylor Mon, 04/12/2021 – … Continue reading →
As an autologous cell therapy, manufacturing the infusion product is a highly specialised process involving tumour harvest and the extraction and large-scale ex vivo expansion of TILs. Tumour-infiltrating lymphocyte (TIL) therapy has been investigated as a treatment modality for melanoma for almost 20 years. versus 21.4% respectively.
California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).
Diurnal in vivo xylem sap glucose and sucrose monitoring using implantable organic electrochemical transistor sensors Credit: Thor Balkhed Researchers at Linköping University, Sweden, have developed biosensors that make it possible to monitor sugar levels in real time deep in the plant tissues – something that has previously been impossible.
oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. . These programmes will include therapies and vaccines in infectious disease and oncology areas.
Editas Medicine to Present Preclinical Data Demonstrating Advancements in In Vivo Gene Editing Approach for the Treatment of Genetic Ocular Diseases at the Association for Research in Vision and Ophthalmology Annual Meeting Editas Medicine to Present Preclinical Data Demonstrating Advancements … Continue reading →
The two partners will take an in vivo approach to the problem of so-called sensorineural hearing loss (SNHL) looking at ways to replace sound-detecting hair cells in the inner ear (cochlea) that become damaged in this type of deafness. An estimated 1.57 Astellas is already working on other approaches to treating hearing loss.
Under the agreement, Sanofi will receive non-exclusive rights to Scribe’s CRISPR by Design platform of wholly-owned enzymes for developing ex vivo NK cell therapies. The alliance will leverage the CRISPR genome editing technologies of Scribe to facilitate in genetic modification of new natural killer (NK) cell therapeutics for cancer.
Martin Borch Jensen to present new research in the biology of aging at the world’s largest aging research for drug discovery conference Credit: Insilico Medicine Hong Kong Limited Wednesday, June 23, 2021, London — Martin Borch Jensen, PhD.,
CytoMed Therapeutics has signed a research collaboration deal with the University of Texas MD Anderson Cancer Center (MD Anderson) in the US to use gamma-delta T cells (gdTc) to treat acute myeloid leukaemia (AML) and breast cancer. MD Anderson leukaemia associate professor Venkata Lokesh Battula will lead the research study.
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.
Moderna Research and Early Development senior vice-president Rose Loughlin said: “Moderna continues to invest in innovative technology to enable us to develop a breadth of transformative medicines for patients. Both companies will use the partnership for advancing in vivo immune cell targeting as a new class of genetic medicines.
In vivo and in vitro pre-clinical studies demonstrated that CFT8919 provides wider coverage of on-target resistant mutations and intracranial activity, indicating its potential to be effective against brain metastases. The agreement will cover the Hong Kong special administrative region (SAR), the Macau SAR and Taiwan.
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