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.
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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.
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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pharmaphorum
SEPTEMBER 15, 2022
Yesterday, the biotechnology company Capstan Therapeutics launched with $165 million in seed and Series A funding for it to build on its foundational insights into precision in vivo engineering technology. Our ambition at Capstan is to invent new clinical paradigms through targeted in vivo reprogramming of cells.
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.
Pharmaceutical Technology
MAY 17, 2023
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. Scribe will receive $75m, including an upfront payment and equity investment via a convertible note.
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.
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
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.
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.
pharmaphorum
JUNE 28, 2021
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.
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 →
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).
Drug Discovery World
MAY 15, 2024
In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. The in vivo data showed statistically significant higher expression level and durability for circVec 2.1
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 →
Pharmaceutical Technology
AUGUST 18, 2022
The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia thalassemia in adult and paediatric patients. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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.
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.
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.
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 →
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.
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.”
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
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.
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.
Drug Discovery World
DECEMBER 15, 2022
Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. In one example, Choi et al.
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.
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.
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.
Drug Discovery World
MAY 17, 2024
The top stories: Gene therapy cures profound genetic deafness within 24 weeks The investigational gene therapy DB-OTO improved hearing to normal levels in a child (dosed at 11 months of age) within 24 weeks, according to trial data presented at the American Society of Gene and Cell Therapy (ASGCT) annual conference.
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.
WCG Clinical
MAY 28, 2024
CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.
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.
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.
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.
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
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.
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.
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.
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.
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.
Pharmaceutical Technology
FEBRUARY 15, 2023
Innovation S-curve for the pharmaceutical industry Gene splicing using nucleases is a key innovation area in the pharmaceutical industry Nucleases play a fundamental role in the field of recombinant DNA technology, or genetic engineering. Gene splicing using nucleases is used to design gene therapeutics for various genetic disorders.
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).
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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