Lilly dives into gene editing
Bio Pharma Dive
NOVEMBER 20, 2020
Through a deal with Precision Biosciences, the pharma aims to develop therapies for genetic disorders, including Duchenne muscular dystrophy.
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Bio Pharma Dive
NOVEMBER 20, 2020
Through a deal with Precision Biosciences, the pharma aims to develop therapies for genetic disorders, including Duchenne muscular dystrophy.
Bio Pharma Dive
JUNE 15, 2021
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 genetic medicines.
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Worldwide Clinical Trials
NOVEMBER 27, 2023
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.
pharmaphorum
JANUARY 29, 2021
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. First of all it will be used for ultra-rare monogenic disease.
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.
Pharmaceutical Technology
FEBRUARY 23, 2023
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.
Bio Pharma Dive
FEBRUARY 28, 2024
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 genetic medicines.
STAT News
FEBRUARY 22, 2023
Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.
Pharmaceutical Technology
JUNE 15, 2023
Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). The company will receive a combined upfront payment and equity investment of $60m.
Worldwide Clinical Trials
NOVEMBER 12, 2024
CGTs address rare and complex diseases at the root cause with increasing use in cancer, genetic disorders, and autoimmune diseases. Despite their exciting potential, the smooth operation of cell therapy development trials requires extraordinary orchestration, perfectly aligning the product and patient journeys.
Camargo
JULY 27, 2021
How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, 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. Formulation Considerations.
XTalks
DECEMBER 14, 2022
Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.
Pharmaceutical Technology
MAY 25, 2023
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. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.
Pharmaceutical Technology
MARCH 28, 2023
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.
Pharmaceutical Technology
JUNE 8, 2023
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.
Scienmag
MAY 2, 2022
WINSTON-SALEM, NC – May 2, 2022 — Wake Forest Institute for Regenerative Medicine (WFIRM) scientists working on CRISPR/Cas9-mediated gene editing technology have developed a method to increase efficiency of editing while minimizing DNA deletion sizes, a key step toward developing gene editing therapies to treat genetic diseases.
pharmaphorum
SEPTEMBER 28, 2022
Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.
pharmaphorum
OCTOBER 4, 2022
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.
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. Further back in development are drugs that will be administered to edit genes within the body, but the first of these candidates are now in clinical trials.
Pharmaceutical Technology
FEBRUARY 13, 2023
Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. Meanwhile, others are researching the link between gene variation and different responses to treatments.
BioPharma Reporter
FEBRUARY 23, 2023
The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.
Scienmag
AUGUST 18, 2020
Credit: Rasgon Lab, Penn State Researchers who developed an improved method of gene editing for the study of arthropods will expand the technology for use in vertebrate species such as mice, fish and birds after receiving new funding from the National Science Foundation.
pharmaphorum
AUGUST 24, 2021
Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 If approved, it could mount a challenge to bluebird bio’s gene therapy Zynteglo, which is already approved in Europe.
STAT News
SEPTEMBER 20, 2022
A new CRISPR startup — backed by some big names in venture capital — is planning to develop gene-editing treatments that can insert a genetic sequence of any length, at any location in the DNA strand, according to industry insiders and documents.
pharmaphorum
OCTOBER 13, 2022
Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. Novartis also recently inked an up-to $1.5
pharmaphorum
JUNE 23, 2022
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). billion in potential milestones if the project advances through development and onto the market. billion agreement that started in 2020.
Scienmag
MAY 26, 2022
A line of powerful tools has been developed from the popular CRISPR-Cas9 to cure genetic diseases. ITHACA, N.Y. — CRISPR has ushered in the era of genomic medicine.
Worldwide Clinical Trials
OCTOBER 24, 2023
While this is a small number of people in each rare disease patient community, the significant and continued growth of rare disease drug development is unsurprising given that there are over 10,000 rare diseases , with more being discovered every year. Roughly 80% of rare diseases are due to a known genetic driver.
pharmaphorum
APRIL 23, 2021
Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt.
pharmaphorum
FEBRUARY 3, 2021
Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.
XTalks
AUGUST 29, 2022
Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome.
STAT News
NOVEMBER 4, 2022
A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. The clinical trial was to be the first time anyone got a gene editing therapy for muscular dystrophy.
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.
Pharmaceutical Technology
MARCH 2, 2023
QN-019a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia). QN-019a overview QN-019a is under development for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL).
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.
BioPharma Reporter
AUGUST 30, 2021
Boston-based company, Vertex, has signed a deal to use Arbor Biotechnologiesâ CRISPR gene-editing technology to develop novel cell therapies for the treatment of serious diseases.
pharmaphorum
APRIL 23, 2021
CRISPR Therapeutics is to receive a hefty $900m payment from Vertex after the companies amended a collaboration to develop, manufacture and market a gene editing therapy for sickle cell disease and beta thalassemia.
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.
XTalks
DECEMBER 20, 2023
As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. Regulatory bodies are also taking note of the applications of AI in drug development.
pharmaphorum
OCTOBER 21, 2020
million) to develop its gene therapy for a rare ocular disease. In a statement, the company said funds will be used to further develop SparingVision’s SPVNo6, for treating all forms of the rare eye disease retinitis pigmentosa. There is currently no treatment approved for RP patients independently of their genetic background.
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. (..)
Scienmag
APRIL 13, 2021
Credit: Copyright © 2021, Kunihiro Shiomi, Shinshu University Diapause is a phenomenon in which animals and insects foresee changes in the environment and actively reduce metabolism, or halt regular differentiation and development. By […].
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.
pharmaphorum
FEBRUARY 16, 2022
She received a cord blood transplant to treat the AML, receiving stem cells carrying a genetic mutation (CCR?5/?32) Having a third functional cure of HIV nevertheless does give pointers to how a more widely applicable cure might be developed in the future, for example using gene-editing drugs to introduce CCR5?32
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