Scribe and Sanofi expand genetic therapy development deal
Pharmaceutical Technology
JULY 18, 2023
Scribe Therapeutics and Sanofi have expanded partnership to progress the development of in vivo genetic therapies to treat genomic diseases.
‘In vivo’ cell therapy: expanding beyond CAR-T
Bio Pharma Dive
OCTOBER 18, 2022
At least five startups have emerged with new ways to genetically modify immune cells within the body, an approach that, if successful, could widen the field of CAR-T treatment.
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Regeneron, Intellia target neurological diseases in expanded gene editing deal
Bio Pharma Dive
OCTOBER 3, 2023
The longtime partners believe that, by combining their technologies, they can create “in vivo” genetic medicines for nervous system and muscular disorders.
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Moderna and Generation Bio partner for genetic medicines development
Pharmaceutical Technology
MARCH 24, 2023
Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.
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Single-course in vivo base editing therapy proven to lower cholesterol
Drug Discovery World
NOVEMBER 14, 2023
FH is one of the most common genetic conditions, affecting around one in 300 people globally. Verve-101 is an investigational, in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood LDL-C. “Of
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Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug
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. ATTR amyloidosis is a rare, progressive and fatal disease.
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Researchers reverse the in vitro and in vivo effects of the mutation that causes Stormorken syndrome
Medical Xpress
MAY 16, 2023
They study gene variation and mutations that cause rare genetic diseases. A mutation is a permanent change in the genetic material. 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.
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New Preclinical Data Presented at the 2021 American Society of Genetic & Cell Therapy Annual Meeting Highlights Precision BioSciences’ ARCUS In Vivo Genome Editing
BioTech 365
MAY 11, 2021
New Preclinical Data Presented at the 2021 American Society of Genetic & Cell Therapy Annual Meeting Highlights Precision BioSciences’ ARCUS In Vivo Genome Editing New Preclinical Data Presented at the 2021 American Society of Genetic & Cell Therapy Annual Meeting … Continue reading →
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Start-up aims to advance transformative genetic medicines
Drug Discovery World
FEBRUARY 16, 2023
Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. The post Start-up aims to advance transformative genetic medicines appeared first on Drug Discovery World (DDW).
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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
BioTech 365
MAY 1, 2021
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 →
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Moderna seals pact with Life Edit Therapeutics to tackle genetic diseases with mRNA-based gene editing
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.
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Eligo raises $30 million to further gene editing within the microbiome
Drug Discovery World
DECEMBER 6, 2023
“We are impressed by the unique delivery and editing technologies that Eligo has developed, and we believe this will set the stage for Eligo to forge new paths in the application of in vivo gene-editing technologies.” The investment will fuel pre-IND and IND activities to achieve early human data readouts in a PhIb/IIa clinical trial.
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The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment
Worldwide Clinical Trials
NOVEMBER 27, 2023
Due to inherited genetic mutations, the red blood cells of patients with severe sickle cell disease become misshaped and jagged, leading to obstructed blood vessels, intense pain, damaged organs, and other serious complications such as stroke. In June 2023, the U.S.
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Scribe Therapeutics Unveils Fully Integrated Platform for Engineering Breakthrough CRISPR-based Genetic Medicines
BioTech 365
OCTOBER 6, 2020
Built by CRISPR leaders including Jennifer Doudna to enable best-in-class in vivo therapeutics that permanently treat the underlying cause of disease Initial $20 million Series A led by Andreessen Horowitz Collaboration with Biogen Inc. focused on development of novel genetic … Continue reading →
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STAT+: Intellia says CRISPR treatment safely corrects DNA of six patients with rare disease
STAT News
SEPTEMBER 16, 2022
Intellia Therapeutics said Friday the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells. Participants in the high-dose group have not yet completed the 16-week observation period.
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Moderna partners with Life Edit for mRNA gene editing therapies
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. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”
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Collaboration boosts development of novel in-situ CAR-T cell therapies
Drug Discovery World
JANUARY 5, 2024
In non-human primate studies, the therapy has demonstrated effective, durable, and well tolerated in vivo CAR-T cell generation. In non-human primate studies, the therapy has demonstrated effective, durable, and well tolerated in vivo CAR-T cell generation.
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Vertex eyes controllable genetic drugs with $1.3bn Obsidian alliance
pharmaphorum
APRIL 23, 2021
billion in milestone payments in the deal, which will focus on using small-molecule compounds to “fine tune” the effects of genetic medicines in the body. Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt. Vertex is also offering up to $1.3
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Evotec and Variant Bio partner to develop fibrosis treatments
Drug Discovery World
APRIL 24, 2024
Evotec will identify best-in-class small molecules targeting a key fibrotic pathway with strong genetic support identified by Variant Bio and progress the programme towards the selection of a clinical development candidate(s) using its R&D platform.
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Astellas teams up with UK biotech Mogrify on hearing loss project
pharmaphorum
JULY 5, 2022
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.
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Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem
Pharma Marketing Network
DECEMBER 21, 2020
Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.
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Precision Bio climbs on near-$1.5bn sickle cell pact with Novartis
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). 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.
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Reaction Biology to acquire Vyant Bio subsidiary’s operations in US
Pharmaceutical Technology
NOVEMBER 4, 2022
The staff in the US from the acquired business will be retained by Reaction to set up its presence in Hershey, which comprises in vitro and in vivo expertise. The staff in the US from the acquired business will be retained by Reaction to set up its presence in Hershey, which comprises in vitro and in vivo expertise. at closing.
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Ginkgo acquires machine learning biotech Patch Biosciences
Drug Discovery World
MARCH 7, 2024
Ginkgo is also working with Sensible Biotechnologies to develop an in vivo microbial mRNA manufacturing platform. Additionally, Ginkgo announced the successful completion of a manufacturing optimisation program with Biogen, focused on redefining the industry standard for manufacturing recombinant adeno-associated virus (AAV)-based vectors.
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Scribe and Sanofi partner to develop cell therapies for cancer
Pharmaceutical Technology
SEPTEMBER 29, 2022
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. 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.
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Merck and Orna partner for RNA technology-based vaccines and therapies
Pharmaceutical Technology
AUGUST 17, 2022
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.
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KSQ and Takeda expand partnership for new tumour-intrinsic targets
Pharmaceutical Technology
MAY 18, 2023
Takeda oncology drug discovery unit head Kathy Seidl stated: “We are encouraged by KSQ’s CRISPRomics platform and its ability to perform in vivo genetic screens for the discovery and validation of tumour targets, which have the potential to modulate the innate and adaptive immune system. “We
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Bayer trumpets $1bn CRISPR deal with Mammoth Bio
pharmaphorum
JANUARY 10, 2022
The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for liver-targeted diseases. – are around one-third the size of the more commonly used Cas9.
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Research partners advance AAV gene therapy for heart disease
Drug Discovery World
AUGUST 7, 2023
Together, Evox and Icahn Mount Sinai are focused on tackling a long-standing challenge in cardiovascular medicine, namely the safe and effective delivery of genetic medicines to cardiomyocytes.
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Sanofi acquires Tidal Therapeutics, adding innovative mRNA-based research platform with applications in oncology, immunology, and other disease areas
The Pharma Data
APRIL 9, 2021
Sanofi today acquired Tidal Therapeutics, a privately owned, pre-clinical stage biotech company with a novel mRNA-based approach for in vivo reprogramming of immune cells. Tidal Therapeutics utilizes a novel mRNA-based approach to in vivo reprogramming of immune cells. About Tidal Therapeutics.
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Leading innovators in transgenic murine models for the pharmaceutical industry
Pharmaceutical Technology
MARCH 9, 2023
Innovation S-curve for the pharmaceutical industry Transgenic murine models is a key innovation area in pharmaceutical s Transgenic murine models refer to mice that have been genetically altered for the purposes of understanding the in vivo functions of genes.
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Gene editing: beyond the hype
pharmaphorum
JANUARY 29, 2021
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).
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High risk, high reward – the future of CAR-T therapy in CLL
Pharmaceutical Technology
OCTOBER 5, 2022
Autologous CAR-T therapy does not easily lend itself to treating CLL, as it relies on extracting functional T-cells from the patient’s blood and genetically modifying them in vitro. Autologous CAR-T agents are now becoming the standard of care for patients in the third line and later settings, providing lucrative returns for these agents.
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HDAC6 can control tumor growth and halt metastasis in triple-negative breast cancer
Scienmag
AUGUST 5, 2020
5, 2020) — Genetic modifier HDAC6 was found to control tumor growth and halt metastasis in triple-negative breast cancer in vivo, according to a new study published […].
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First non-human primate study showing promise of gene therapy for stroke repair
Scienmag
NOVEMBER 12, 2020
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. Credit: Jinan University Stroke is a leading cause of death and severe long-term disability with limited treatment available.
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Ixaka (formerly Rexgenero) Launches as an Integrated Cell and Gene Therapy Company
The Pharma Data
JANUARY 17, 2021
Joe Dupere, CEO of Ixaka, commented: “Ixaka’s broad offering of integrated cell and gene therapy capabilities, encompassing cell-based products and an innovative in vivo gene delivery platform, provides a strong foundation for our ambitions to become a leader in cell and gene therapies. A total of $15.4
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A step closer to ‘clinical trial in a dish’ for Alzheimer’s
Drug Discovery World
JUNE 8, 2023
sAD has no specific family link and is caused by a combination of genetics, the environment and a person’s lifestyle. sAD has no specific family link and is caused by a combination of genetics, the environment and a person’s lifestyle.
Closing the translation gap in oncology drug development
Drug Discovery World
MAY 9, 2023
The recent passage of the FDA Modernization Act, which removed the requirement for animal testing when suitable alternatives are available, has highlighted the inadequacy of animal models most often used for in vivo research. Cancer is far more complex than the sum of its parts. Mice have long been heroes of preclinical cancer research.
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€2m raised in seed funding to develop novel therapies for Cystic Fibrosis
Drug Discovery World
JUNE 19, 2023
Anoat will use the funds to conduct additional in vivo and in vitro pharmacology assessments and select a lead candidate. Cystic fibrosis is a progressive genetic disease that affects the lungs, pancreas and other organs.
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LNP formulations produce strong immune responses, data shows
Drug Discovery World
FEBRUARY 15, 2024
The etherna paradigm for cLNP design is ushering in the rapid generation of novel families of delivery systems for not only vaccination but also for in vivo tolerisation for a host of autoimmune diseases.” The post LNP formulations produce strong immune responses, data shows appeared first on Drug Discovery World (DDW).
Sana Bio’s $150 Million IPO Expected to Provide Market Valuation of $10 Billion
The Pharma Data
JANUARY 17, 2021
Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.
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A CNIO team describes how a virus can cause diabetes
Scienmag
OCTOBER 20, 2020
Credit: CNIO It has recently been described that infection by some enteroviruses – a genus of viruses that commonly cause diseases of varying severity – could potentially trigger diabetes, although its direct effect ‘in vivo’ as well as its mechanism of action at the molecular level were unknown.
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Precision BioSciences Announces Executive Leadership Change
The Pharma Data
DECEMBER 13, 2020
Looking ahead, our top priority remains focused on advancing our allogeneic CAR T cell therapy and in vivo gene correction pipelines to speed development of medicines for unmet needs in cancer and rare diseases.”. About Precision BioSciences, Inc. Precision BioSciences, Inc.
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