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.

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Gene Editing In-Vivo Gene Sequencing Gene 97

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Pharmaceutical Technology

FEBRUARY 23, 2023

DNA binding site prediction, peptide structure optimisation, and AI-assisted genome analysis are some of the accelerating innovation areas, where adoption has been steadily increasing. However, not all innovations are equal nor do they follow a constant upward trend.

Drugs 189

Drugs DNA In-Vivo In-Vitro 189

XTalks

AUGUST 5, 2020

Single cell RNA sequencing technology has been extensively applied to understand heterogeneity among tumor cells, within the tumor microenvironment and during cell development. Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level. The Power of Single Cell Technology.

Gene Expression 112

Gene Expression Development RNA Drugs 112

Pharmaceutical Technology

FEBRUARY 15, 2023

They are engineered to cut specific genomic targets in order to modify the expression of single genes and proteins. According to GlobalData, there are 190+ companies, spanning technology vendors, established pharmaceutical companies, and up-and-coming start-ups engaged in the development and application of gene-splicing using nucleases.

Gene Splicing 130

Gene Splicing Gene DNA Recombinant DNA Technology 130

Scienmag

NOVEMBER 16, 2020

Wnt-regulated lncRNA discovery enhanced by in vivo identification and CRISPRi functional validation. Genome Med 12, 89 (2020). Singapore scientists uncover potential role of long non-coding RNAs in pancreatic cancer Credit: From Figure 4 in Liu, S., Harmston, N., Glaser, T.L.

In-Vivo 53

In-Vivo RNA DNA Genome 53

Roots Analysis

FEBRUARY 27, 2024

The global genome editing market is anticipated to grow at a CAGR of 12.6% The global genome editing market is anticipated to grow at a CAGR of 12.6% The biopharmaceutical pipeline includes close to 500 gene therapies , several of which are being developed based on CRISPR technology. during the forecast period 2023-2035.

Genome 40

Genome Genomics Gene In-Vivo 40

Pharmaceutical Technology

FEBRUARY 14, 2023

These systems include human and mouse cell lines, and even in vivo in live animals. These systems include human and mouse cell lines, and even in vivo in live animals. However, not all innovations are equal and nor do they follow a constant upward trend.

In-Vivo 130

In-Vivo DNA Antibody Gene 130

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.

Life Science 119

Life Science Gene Editing Development Clinical Trials 119

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

Gene Editing 98

Gene Editing Trials Drugs In-Vivo 98

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.

In-Vivo 130

In-Vivo Antibody Genetics Gene 130

The Pharma Data

DECEMBER 13, 2020

Nasdaq: DTIL) a clinical stage biotechnology company dedicated to improving life with its novel and proprietary ARCUS® genome editing platform, today announced that Abid Ansari, Chief Financial Officer, notified the Company that he will be leaving the organization after nearly five years to pursue a new career opportunity. “On DURHAM, N.C.,

In-Vivo 52

In-Vivo Genome Genomics Gene 52

The Pharma Data

JANUARY 12, 2021

Working alongside KSQ will facilitate smart drug discovery and development of what we hope will be transformative new therapies for patients with intractable forms of cancer.”. In addition, KSQ will be eligible for additional option payments along with development and commercialization milestone payments. sales for that product.

Development 52

Development Research In-Vivo Genome 52

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., as Head of Business Development. .–( BUSINESS WIRE )– EdiGene, Inc. as Head of the US Subsidiary, and Kehua Fan, M.D.,

Business Development 40

Business Development Development Gene Editing In-Vivo 40

The Pharma Data

DECEMBER 2, 2020

” VANCOUVER, BC, December 02, 2020 /24-7PressRelease/ — Eyam Vaccines and Immunotherapeutics (EYAM) today announced that former President and CEO of Genome Prairie is joining EYAM. Pontarollo’s primary areas of research and technical expertise include Genomics, Molecular Biology, Vaccine development and Immunology.

Vaccine 52

Vaccine Vaccination Genome Genomics 52

pharmaphorum

OCTOBER 6, 2022

A complex range of factors are thought to contribute to the development of the illness. After developing a conventional disease pathway model for GWI, researchers then used Elsevier’s data and technology to create a model to act as a basis for in silico modelling of the disease. Disease modelling Gulf War Illness.

In-Vivo 98

In-Vivo Research Genetics Drugs 98

XTalks

MAY 22, 2024

In this episode, Ayesha spoke with John Finn, PhD, Chief Scientific Officer at Tome Biosciences , a company developing programmable gene insertion (PGI) technology. PGI is a revolutionary approach for the development of potentially curative cell and integrative gene therapies.

Gene Editing 52

Gene Editing Life Science Gene Gene Therapy 52

The Pharma Data

JANUARY 10, 2021

Nasdaq: EDIT), a leading genome editing company, today announced the U.S. The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial. EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

pharmaphorum

OCTOBER 8, 2020

Scribe – which counts new Nobel Prize for chemistry winner Jennifer Doudna among its founders – is getting $15 million upfront from Biogen for an alliance focusing on the development of a CRISPR-based therapy for neurodegenerative disease including amyotrophic lateral sclerosis (ALS). Benjamin Oakes.

Gene Editing 69

Gene Editing Gene In-Vivo Clinical Trials 69

The Pharma Data

NOVEMBER 22, 2020

Peer stated that his team plans to develop a treatment for all cancers and that their technique could be ready for humans within the next two years. CTX001 is being developed under a co-development and co-commercial agreement between the two organizations. Photo courtesy of Science Advances.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

The Pharma Data

JUNE 7, 2023

a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics. Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver.

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Gene Therapy Gene Gene Editing In-Vivo 40

Delveinsight

FEBRUARY 25, 2021

Instead of snipping the genome, base editing lets for edits of individual letters in a genetic sequence. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. GuideTx will aid Beam in developing LNP-based medicines. Beam employs three approaches to deliver its genetic medicines to cells.

DNA 52

DNA In-Vivo Genetics Medicine 52

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

The Pharma Data

DECEMBER 1, 2020

NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo , announced today the pricing of an underwritten public offering of 4,794,521 shares of its common stock at a public offering price of $36.50 CAMBRIDGE, Mass.,

In-Vivo 40

In-Vivo Containment Genome Genomics 40

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.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

XTalks

NOVEMBER 3, 2023

CRISPR works as genetic scissors to edit parts of the genome. The scientists on the advisory committee were particularly focused on the CRISPR gene editing technology itself and how companies like Vertex that are developing CRISPR-based therapies are ensuring their treatments are not making off-target gene edits.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

The Pharma Data

OCTOBER 14, 2020

Priothera will use the funds to progress the clinical development of mocravimod, a modulator of sphingosine 1 phosphate (S1P) receptors, to enhance the curative potential of allogeneic hematopoietic stem cell transplantation (HSCT) for treating AML. All jobs have been maintained and the workforce has grown from about 220 to 350.

Clinical Trials 52

Clinical Trials Clinical Trials Manufacturing In-Vivo 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.

Vaccine 52

Vaccine Vaccination In-Vivo Genetics 52

The Pharma Data

DECEMBER 10, 2020

Many people who have been exposed to the “common cold” potentially develop adenovirus immunity: the immune system often attacks and disables these first-generation vaccines before they can activate the immune response to attack the SARS-CoV-2 virus. This blocking of viral replication was observed in both the lung and nasal passages.

Vaccine 52

Vaccine Vaccination Immune Response Protein 52

The Pharma Data

JANUARY 4, 2021

Kiromic chPD1 has shown in preclinical data to show a cytotoxic response in 9 different in vivo models with 100% long-term PFS with the induction of host memory responses. PD-1 has always been a challenge for CAR-T development. “We believe Prof. About PD-1 Check-point inhibition. Dr. Barber is a tumor immunologist.

In-Vivo 40

In-Vivo Development Immune Response Big Data 40

XTalks

AUGUST 12, 2020

The identification of T cells that essentially ‘hide’ the virus has significant implications for developing therapeutic strategies to improve the treatment and long-term management of HIV. This may help explain why HIV is adequately controlled, but not eradicated, with current treatments. The research study was published in Cell Reports.

RNA 52

RNA Protein DNA In-Vivo 52

FDA Law Blog

DECEMBER 6, 2024

Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. academic to industry) involved in developing CGTs, the guidance starts by summarizing some of the fundamentals of opening an IND.

Gene Therapy 59

Gene Therapy Gene Production In-Vivo 59

Roots Analysis

JANUARY 27, 2025

Most of the companies offer protein expression for research applications, due to their extensive use in early-stage discovery, lead candidate screening and in vivo studies. Considering their vast potential, close to 50% of the top selling drugs in 2023 were protein therapeutics.

Protein 40

Protein Reagent Marketing Gene 40