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.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

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. . Merck will also make royalty payments on any approved products developed out of the partnership.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

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. Sanofi will also make tiered royalty payments on net sales of products resulting from the collaboration in the future.

Development 130

Development In-Vivo Genome Genomics 130

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.

Gene Editing 130

Gene Editing Gene Gene Therapy In-Vivo 130

pharmaphorum

APRIL 25, 2022

The UNC and NCSU team, led by biomedical engineer Yevgeny Brudno, report in Nature Biotechnology how they have developed an implant that can generate the CAR-T cells in vivo. In some cases where the cancer is particularly aggressive, that timeframe may simply be too long to be a viable option for the patient.

Engineer 105

Engineer In-Vivo FDA Approval Manufacturing 105

pharmaphorum

OCTOBER 6, 2022

By reducing the disease to its key components, a computer-readable network of disease markers was characterised using genetic, biomarker, and clinical data; the model was also populated with ‘disease vs control’ data. Disease modelling Gulf War Illness. Tinker, tailor, soldier, therapy.

In-Vivo 98

In-Vivo Research Genetics Drugs 98

Roots Analysis

JANUARY 27, 2025

Cell therapies are based on the premise that the patients own cells (autologous), or those from a healthy donor (allogeneic), can be genetically re-programmed to combat various diseases. As a result, the mass production of such products is still a concern. As a result, the mass production of such products is still a concern.

Manufacturing 40

Manufacturing Contamination Production Gene Therapy 40

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 References 1.

Gene Therapy 40

Gene Therapy Gene In-Vivo Genetics 40

pharmaphorum

DECEMBER 6, 2020

The trials are the first to test a CRISPR/Cas9 gene editing therapy in humans for a genetic disease, according to the partners. CTX001 is an ‘ex vivo’ application of gene-editing, in which the technology is used to modify a patient’s own cells outside the body. billion product if it gets approved for both indications.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

XTalks

AUGUST 5, 2020

Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level. Recent advances in the technology have allowed for the interrogation of distinct subsets of cell populations within tissues, and associated molecular markers that may function as important disease drivers.

Gene Expression 112

Gene Expression Development RNA Drugs 112

The Pharma Data

DECEMBER 13, 2020

Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Dong Wei, CEO of EdiGene. Dr. Zhang completed his postdoctoral fellowship at Harvard Medical School/Boston Children’s Hospital.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

The Pharma Data

NOVEMBER 22, 2020

Peer went onto say that he believes that, in the near future, there will be many personalized treatments based on genetic messengers. The product is the most advanced gene-editing approach in development for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease. . Photo courtesy of Science Advances.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

The Pharma Data

NOVEMBER 17, 2020

Dr. Anderson has been an early adopter in blastocyst embryo transfer, cryopreservation and pioneered blastocyst biopsy with and without laser technology for genetic testing. . SARASOTA, Fla. , 18, 2020 /PRNewswire/ — INVO Bioscience , Inc. Drs. and across the world.

In-Vivo 52

In-Vivo In-Vitro Genetics Doctors 52

Roots Analysis

SEPTEMBER 20, 2023

Cell therapies are based on the premise that the patient’s own cells ( autologous ), or those from a healthy donor ( allogeneic ), can be genetically re-programmed to combat various diseases. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit.

Manufacturing 40

Manufacturing Production Contamination Gene Therapy 40

The Pharma Data

DECEMBER 27, 2020

Changes in heparan sulfate (HS) concentration in cerebrospinal fluid (CSF) are being monitored in patients treated with LYS-SAF302 to provide evidence of in vivo biological activity of the drug and demonstrate proof of concept. Average reductions were highly statistically significant. About Lysogene. Sarepta Therapeutics, Inc.

Gene Therapy 40

Gene Therapy Clinical Trials Clinical Trials In-Vivo 40

XTalks

NOVEMBER 3, 2023

CRISPR works as genetic scissors to edit parts of the genome. Related: Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug The advisors appeared convinced that exa-cel can offer significant benefits to people with sickle cell. I think in this case, there is a huge unmet need.”

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. Of all the Angelman syndrome pipeline therapies , the most touted treatment was Gaboxadol ( OV101 or THIP ), a product of Ovid Therapeutics. As a consequence, Ovid halted everything related to OV101 and Angelman syndrome.

Marketing 52

Marketing Gene Drugs Protein 52

Roots Analysis

SEPTEMBER 5, 2023

Further, owing to lack of understanding of how drug molecules were likely to behave in vivo , many lead molecules that progressed into the clinical stages of development had to be discontinued when they failed to demonstrate adequate therapeutic benefit.

Drugs 52

Drugs In-Vivo Development Research 52

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.

In-Vivo 105

In-Vivo Engineer Scientist Life Science 105

XTalks

OCTOBER 1, 2020

Leaving academia for industry is a route that academic scientists and professors did not traditionally pursue in the past due to perceived differences in skillsets, experiences and even associated stigmas. However, this has since changed, and now, transitioning between academia and industry has become more commonplace. Key Moments.

Scientist 52

Scientist In-Vitro Research Life Science 52

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Dr. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

MAY 18, 2023

KSQ will receive future payments of up to $510m on achieving all milestones during the agreement period, along with potential net sales royalties on any commercial product that is the result of the partnership. Takeda will make an upfront payment and provide an investment in the double-digit millions of dollars to KSQ.

Gene Therapy 130

Gene Therapy In-Vivo Gene Genetics 130

XTalks

DECEMBER 13, 2023

While Lyfgenia delivers a functional copy of hemoglobin A to replace the abnormal one in hematopoietic (blood) stem cells via a lentiviral vector, Casgevy edits the HBB gene using CRISPR technology to increase the production of fetal hemoglobin in blood stem cells. Casgevy is also the first ever CRISPR/Cas9-based therapy approved in the US.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

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. Early-stage companies are often years, often a decade or longer, from a marketable product.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

Roots Analysis

SEPTEMBER 18, 2023

Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry. Therefore, in order to store and preserve such enormous amounts of blood, single-use assemblies are a necessity.

Engineer 52

Engineer Containment In-Vivo Production 52

Pharmaceutical Technology

APRIL 12, 2023

Pharmaceutical research has long relied on non-human primate models for early-stage discoveries, but their use continues to cause controversy. This was part of an investigation into an international primate smuggling ring. Globally, non-human primates make up a relatively small percentage of the animals used in research.

Regulation 245

Regulation Medicine Vaccine Vaccination 245

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Gene 52

The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

Roots Analysis

AUGUST 31, 2023

An additional level of genetic control is provided by gene switches that are located upstream of the promoter region. These genetic switches assist transcription factors in binding to the promoter region. Several diseases are caused by sub-optimal gene expression or through uncoordinated or unsynchronized production of a gene product.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

The Pharma Data

JANUARY 10, 2021

EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in HbF production, which has the potential to provide a one-time, durable treatment benefit for people living with sickle cell disease.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Roots Analysis

OCTOBER 19, 2023

Bacteria plays a crucial role in maintaining the ecosystem balance. However, there are few species of bacteria that can cause several infectious diseases ( such as strep throat, salmonellosis, tuberculosis, whooping cough ). These are mainly transmitted through air, water, living organisms, and food.

Bacteria 40

Bacteria Production In-Vivo In-Vitro 40

Roots Analysis

JANUARY 14, 2024

Overview of Biologics Biologics refer to medicinal / therapeutic products that are either manufactured using living organisms or semi-synthesized from biological sources. This can be attributed to the fact that the production of biopharmaceuticals requires living expression systems. Like all drugs, biologics are regulated by the FDA.

Contract Manufacturing 40

Contract Manufacturing Protein Manufacturing Production 40

The Pharma Data

APRIL 8, 2022

The mere motions of breathing are known to influence vital functions of the lungs, including their development in babies, the production of air-exchange-enhancing fluid on their inner surfaces, and maintenance of healthy tissue structure. Every breath stretches the lungs’ tissues with each inhale and relaxes them with each exhale.

Immune Response 52

Immune Response In-Vivo Genetic Analysis Research 52

The Pharma Data

DECEMBER 9, 2020

Vigil’s pipeline strategy is to target these diseases by combining a high-level understanding of microglia physiology, disease genotyping, and patient phenotyping to identify specific genetic variations associated with microglial dysfunction. which will remain a key shareholder. in a statement. . Libra Therapeutics.

Marketing 52

Marketing In-Vivo Protein In-Vitro 52

The Pharma Data

OCTOBER 15, 2020

Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Nielsen, the primary inventor of peptide nucleic acid (PNA) technology, brings extensive experience in genetic medicine to the company.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

Pharma Marketing Network

JANUARY 20, 2021

In other areas of the industry (such as cell and genetic medicine development) progress continued at an impressively brisk pace despite the pandemic. Regarding OWS’s support for vaccine development, Dr Slaoui noted that 5 of the 6 vaccines selected (from a total of 94 programs) are currently in phase 3 development or approved.

Vaccination 52

Vaccination Vaccine In-Vivo Genetics 52

Roots Analysis

AUGUST 23, 2024

Unlike traditional forms of vaccines modern day vaccines use the virus’ genetic code itself to give our cells instruction on how to produce this antigen molecule, meaning we only ever need a tiny amount of an antigen in the cure for it work. Ribosomes are cellular machines that read mRNA sequences and produce proteins.

Protein 40

Protein Vaccination Vaccine Immune Response 40