Drug Discovery World

OCTOBER 10, 2023

The National Institutes of Health has awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome.

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Gene Editing Development Drugs Clinical Trials 52

pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. Scribe has designed, engineered and tested thousands of evolved CRISPR enzymes to build an advanced platform for creating breakthrough in vivo treatments,” said Oakes.

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Gene Editing Gene In-Vivo Clinical Trials 54

Drug Discovery World

MAY 5, 2023

Eterna Therapeutics, a life science company focused on mRNA cell engineering, has acquired Exacis Biotherapeutics’ immuno-oncology platform. The acquisition will provide Eterna with a pipeline of allogeneic immuno-oncology products under development for the treatment of haematologic and solid tumours.

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Gene Editing Engineer DNA Life Science 52

BioPharma Reporter

NOVEMBER 2, 2020

PerkinElmer will acquire UK cell engineering company Horizon Discovery, expanding its portfolio with gene editing and gene modulation tools.

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Gene Editing Engineer Gene Development 98

Pharmaceutical Technology

MARCH 2, 2023

QN-023a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for Relapsed Acute Myeloid Leukemia. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data.

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Gene Editing Genetic Engineering Engineer Clinical Trials 100

XTalks

OCTOBER 20, 2023

Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. NTLA-2001 is the furthest along in testing for Intellia.

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In-Vivo Gene Editing Trials Gene 52

Drug Discovery World

JANUARY 9, 2023

Harvard investigators have developed a new cell therapy approach to eliminate established tumours and induce long-term immunity. . Using gene engineering, we are repurposing cancer cells to develop a therapeutic that kills tumour cells and stimulates the immune system to both destroy primary tumors and prevent cancer.” .

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Drug Discovery World

DECEMBER 8, 2023

ONK Therapeutics and NAYA Biosciences have agreed to develop a combination therapy of ONK’s optimally-engineered natural killer (NK) cell therapies and NAYA’s FLEX-NK bispecific antibodies. Both companies will share the costs of manufacturing and preclinical assessments.

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Antibody Gene Editing Clinical Trials Clinical Trials 52

Drug Discovery World

NOVEMBER 27, 2023

Across the biotherapeutics workflow The landscape of large molecule drug development has evolved significantly in recent decades. Technological innovations supporting discovery and development stages have concentrated on both upstream and downstream aspects of the workflow. This article is sponsored by Revvity.

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Production Engineer Gene Editing Gene 59

Drug Discovery World

FEBRUARY 28, 2024

Lu Rahman selects some of the year’s interesting and noteworthy advances in cancer research drug discovery and development. As always, a year within cancer drug discovery and development brings with it significant breakthroughs, and 2023 has been no exception. Figures from the Amercian Cancer Society 1 state that “a little over 1.9

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Development Research Drugs Antibody 64

Pharmaceutical Technology

APRIL 28, 2023

EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. This increases the expression of fetal haemoglobin.

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Gene Therapy Drugs Gene Gene Editing 264

Pharmaceutical Technology

AUGUST 26, 2022

ElevateBio has signed a long-term strategic collaboration with the University of Pittsburgh, US, to establish a biomanufacturing centre for expediting cell and gene therapy development.

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Gene Therapy Gene Editing Gene Manufacturing 162

pharmaphorum

MAY 7, 2021

Gene editing firm CRISPR Therapeutics has announced a collaboration with US biotech Nkarta to develop natural killer (NK) cell therapies for cancer. It’s at the forefront of gene editing although the technology has spawned rivals including Intellia Therapeutics, Caribou Biosciences, Sangamo Therapeutics and Mammoth Biosciences.

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Gene Editing Gene Genetic Engineering DNA 52

Drug Discovery World

NOVEMBER 1, 2023

For the most part, my job revolves around two things: mentoring my team as they develop as scientists, and communicating what we do ­– making sure that the discoveries we make are shared as widely as possible, so they can have the most value to us, and to the rest of the scientific community.

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Gene Editing Research Gene Genome 59

XTalks

JANUARY 26, 2024

Key breakthroughs at the forefront include novel approaches to cancer treatment and diagnosis, the integration of artificial intelligence (AI) in oncology and the development of advanced preventive measures and care. Overcoming drug resistance: A major challenge in cancer treatment is the development of resistance to chemotherapy drugs.

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Research Gene Editing Gene Genetics 118

Drug Discovery World

OCTOBER 24, 2023

A BioIndustry Association (BIA) report lists the widespread adoption of artificial intelligence (AI) and machine learning (ML), the advancement of gene editing technologies, the power of engineering biology, and a growing interest from major pharmaceutical players as key trends driving the sector.

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Gene Editing Engineer Clinical Trials Clinical Trials 52

Scienmag

JULY 2, 2021

A newly developed CRISPR-Cas9-based tool carries out efficient and long-term gene silencing by epigenetic editing Credit: BioDesign Research Originally discovered as a bacterial mode of defense against invading viruses, the remarkable ability of CRISPR-Cas9 to modify specific locations of DNA has made it a researcher favorite among gene editing tools. (..)

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Gene Silencing Gene Editing Gene DNA 75

Drug Discovery World

JUNE 22, 2023

Evox Therapeutics has purchased Codiak Biosciences engEx-AAV technology platform, including all intellectual property rights, and exclusive access to certain engineering and manufacturing rights. The post Evox Therapeutics acquires exosome AAV technology from Codiak appeared first on Drug Discovery World (DDW).

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Gene Therapy Gene Editing Drug Delivery Gene 52

Scienmag

FEBRUARY 23, 2021

CRISPR gene editing has transformed research, but it is not perfect, and can sometimes target unintended genes; to watch CRISPR enzymes respond to different genes, Leipzig University researchers developed a new method using DNA origami and were able to me Credit: Image courtesy of Julene Madariaga Marcos.

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DNA Gene Gene Editing Scientist 56

Pharmaceutical Technology

JANUARY 10, 2023

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.

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Gene Editing Gene Gene Therapy Genetics 130

Drug Discovery World

DECEMBER 12, 2022

DDW highlights the most interesting global news and comments within the drug discovery and development sector this month. . Everyone enjoys a good news story and the drug discovery and development sector is no exception. Lately, there has been a lot of interest in microbiome research. Analysis . And finally… .

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Drugs Gene Editing Genome Genomics 52

The Pharma Data

JANUARY 24, 2021

a biotechnology company developing renewable stem cell-derived T cell therapies for cancer and other immune disorders, announced today the appointment of Chris Bond , Ph.D. Dr. Bond joins Notch from Kite, where he most recently served as Vice President of Cellular Engineering. . VANCOUVER, BC , Jan.

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Engineer Gene Editing Genome Genomics 52

Drug Discovery World

JANUARY 12, 2023

Oksana Sirenko looks at the advantages and limitations of using organoids from human cell sources in drug development and drug screening. . The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. What are organoids? .

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Gene Editing Gene Drugs In-Vivo 98

Drug Discovery World

SEPTEMBER 6, 2022

As researchers and developers work to transform the way we treat diseases, cell and gene therapies will continue to be the centre of innovation in the coming years. . ViaCyte, Beam, and Verve are all due to begin gene editing trials this year. The future of cell and gene therapy: opportunities for innovation .

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Gene Therapy Gene Gene Editing Manufacturing 52

Drug Discovery World

JULY 3, 2023

This was centred around pre-clinical models (in vitro and in vivo) to support discovery and development, re-entering the fray with costimulatory agonists, and clinical development strategies for combinations with immune cell engagers​.

Antibody 52

Antibody In-Vitro Engineer In-Vivo 52

Drug Discovery World

DECEMBER 20, 2022

While the development of clinical models using AI will continue, there will also be exploration of its ability to increase efficacy and efficiency in diagnostics, telemedicine, clinical trial design, patient engagement and clinician decision support. . AI will take on a wider role.

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Drugs Gene Editing Clinical Trials Clinical Trials 52

Drug Discovery World

JULY 18, 2023

A pioneering scientific site, strategically located At the Cambridge site, Revvity delivers cell line engineering and screening services to improve the outcomes of drug development through enhanced target selection and pre-clinical screening.

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Genome Genomics Life Science Gene Editing 52

pharmaphorum

AUGUST 25, 2022

A new era of cell therapy has well and truly arrived with the iPSC field setting a trailblazing fire with its potential to develop fully scalable, off-the-shelf cell lines reducing the cost of goods and increasing accessibility to patients. Sana Biotechnology , Takeda and many more.

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Gene Editing Genome Genomics Engineer 40

XTalks

MARCH 28, 2024

Colorectal Cancer: Advances in Treatment Options In recent years, there has been considerable progress in understanding the biology of CRC, which has helped develop more effective and personalized treatment options. An overview of some of the most promising developments in CRC treatment is provided below.

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Gene Editing Life Science Clinical Trials Clinical Trials 59

pharmaphorum

JULY 1, 2021

Synthetic biology holds much promise for the life sciences sector as it continues to look for new ways of treating disease and accelerating drug development, says Debiopharm’s Tanja Dowe. There are some big changes coming to pharma and the development of new pharmaceuticals will no longer be the sole provenance of chemists.

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Gene Editing Engineer Development Gene 75

pharmaphorum

SEPTEMBER 28, 2022

Roche’s Genentech division has formed an R&D partnership with Arsenal Biosciences focusing on developing and refining the biotech’s programmable T cell-based therapies platform. Both companies will use the fruits of their collaboration in the development of future therapeutic candidates, said ArsenalBio in a statement.

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Gene Editing Genome Genomics Engineer 57

Cloudbyz

MARCH 6, 2024

The Evolution of Pharmaceutical Therapies: The emergence of biologics represents a significant paradigm shift in the pharmaceutical industry, prompting a reevaluation of traditional drug development and manufacturing processes. Challenges and Future Directions: Despite their immense promise, biologics are not without challenges.

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Gene Immune Response Manufacturing Gene Therapy 73

The Pharma Data

SEPTEMBER 1, 2021

With a goal of developing rheumatoid arthritis therapies with minimal side effects, researchers at Washington University School of Medicine in St. Louis have genetically engineered cells that, when implanted in mice, will deliver a biologic drug in response to inflammation. million adults in the United States.

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Drugs Engineer Drug Delivery Gene 52

pharmaphorum

DECEMBER 21, 2022

Gilead Sciences has bolstered its position in the development of next-generation cancer cell therapies with a deal to absorb Tmunity Therapeutics into its Kite unit. Kite will continue to provide funding to Penn, with the arrangement extended until 2026 once the deal goes through, expected in the first quarter of 2023.

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Gene Editing Licensing Licensing Manufacturing 52

XTalks

NOVEMBER 30, 2023

Global entities such as the WHO, the Global Fund and the Joint United Nations Programme on HIV/AIDS (UNAIDS) have developed comprehensive strategies aligned with Sustainable Development Goal (SDG) target 3.3, These findings signify a significant step forward in the development of AGT103-T as a potential gene therapy for HIV.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

JULY 14, 2022

The research team has created the most detailed map to date of complex gene networks for three important immune genes involved in the role of T-cell function: IL2RA, IL-2 and CTLA4. The maps provide valuable insight into immune cell function and the development of autoimmune diseases. Impact for Patients.

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Gene Regulation Protein Gene Editing 98

Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Gene Editing Gene In-Vivo Genome 52