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). It is also working with Eli Lilly on in vivo gene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4

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Gene Editing In-Vivo Gene Sequencing Gene 97

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.

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Gene Gene Editing Gene Therapy Clinical Trials 130

pharmaphorum

OCTOBER 7, 2020

Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. There is enormous power in this genetic tool, which affects us all. What started as a curiosity?driven,

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Gene Editing Bacteria DNA Gene 105

Pharmaceutical Technology

MARCH 2, 2023

According to GlobalData, Phase I drugs for Relapsed Acute Myeloid Leukemia have a 71% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how QN-023a’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks.

Gene Editing 100

Gene Editing Genetic Engineering Engineer Clinical Trials 100

STAT News

SEPTEMBER 16, 2022

Enjoy, and see you soon … Intellia Therapeutics reported that 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 , STAT writes. Continue to STAT+ to read the full story…

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Gene Editing Genetic Disease Genetics Protein 94

Pharmaceutical Technology

MARCH 2, 2023

According to GlobalData, Phase I drugs for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia) have a 90% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. The company develops gene-editing organ transplantation technology. Buy the report here.

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Gene Editing Clinical Trials Clinical Trials Genetics 100

XTalks

MARCH 7, 2024

In a pivotal move for the food industry, the US Food and Drug Administration (FDA) has unveiled industry guidance for genome edited foods derived from plants. The FDA’s recent guidance document sheds light on the regulatory landscape for foods derived from genome edited plants.

Gene Editing 52

Gene Editing Gene Genomics Genome 52

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. Overcoming drug resistance: A major challenge in cancer treatment is the development of resistance to chemotherapy drugs.

Research 118

Research Gene Editing Gene Genetics 118

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. The post Nobel winner Charpentier’s CRISPR Therapeutics gets $900m in reworked Vertex deal appeared first on.

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Gene Editing Gene DNA Bacteria 64

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

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

pharmaphorum

MARCH 11, 2021

Vertex Pharma has made its name with treatment for cystic fibrosis, but has big plans in cell and genetic therapies – and has just chalked up fast-track status from the FDA for a cell-based therapy for diabetes. . We have never been in more disease areas than we are right now.

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Gene Editing Insulin Immune Response Gene 98

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.

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Gene Editing Gene Genetic Engineering DNA 52

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Pharmaceutical Technology

MARCH 2, 2023

ImmunoGen has announced an agreement granting rights to Vertex Pharmaceuticals to carry out research using antibody-drug conjugate (ADC) technology for the discovery of new targeted conditioning agents. The research using ImmunoGen’s technology will enable the discovery of the agents for use with gene editing.

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Gene Editing Gene Development Research 130

pharmaphorum

FEBRUARY 17, 2022

Global Blood Therapeutics’ run of positive regulatory news for its oral sickle cell disease treatment Oxbryta has continued with an approval for the drug in Europe for patients aged over 12. The post GBT claims EU approval for sickle cell drug Oxbryta appeared first on.

Drugs 59

Drugs Gene Therapy Gene Editing Gene 59

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. There is another $1 billion in downstream payments on offer, assuming the drug candidates all make it through to regulatory approval and hit sales targets.

In-Vivo 52

In-Vivo Gene Editing Gene Therapy Gene 52

pharmaphorum

FEBRUARY 23, 2022

In this context, the 2 nd Annual Gene Therapy Patient Engagement Summit will unite patient advocacy and engagement leaders from gene therapy and gene editing drug developers, patient groups and patients themselves to address the specific challenges of genetic therapy patient engagement, generating actionable insights through detailed case studies (..)

Gene Therapy 52

Gene Therapy Gene Gene Editing Clinical Trials 52

XTalks

MAY 22, 2023

Related: 5 Food Companies Working With Precision Fermentation Technology How Pairwise Leverages CRISPR Technology CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technique that can be used to alter the DNA of cells to enhance certain characteristics or reduce less desirable ones.

Branding 98

Branding Gene Editing DNA Production 98

pharmaphorum

NOVEMBER 1, 2022

Investigating the next generation of genetic medicine through RNA based therapies. RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious, and chronic diseases. Director, Genetic Medicine. Website: [link].

RNA 52

RNA Genetics Gene Editing In-Vitro 52

XTalks

JULY 14, 2022

A significant disruption to these networks can alter the expression of immune genes and result in disease. Mapping networks helps us understand the impact of a disruption and its effect throughout the network, or how a drug can impact numerous proteins at once.

Gene 98

Gene Regulation Protein Gene Editing 98

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

The Pharma Data

SEPTEMBER 1, 2021

Louis have genetically engineered cells that, when implanted in mice, will deliver a biologic drug in response to inflammation. The approach allows those cells to remain in the body for a long time and secrete a drug whenever there is a flare of inflammation.” million adults in the United States.

Drugs 52

Drugs Engineer Drug Delivery Gene 52

pharmaphorum

APRIL 20, 2022

The potential to develop treatments with CRISPR gene editing is hinged on its ability to add, delete or replace elements within a target DNA sequence. Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. Why cancer? The pipeline.

Gene Editing 52

Gene Editing DNA Gene Genetics 52

The Pharma Data

JULY 28, 2021

Novel small molecule drug candidates will target GPR75 to potentially address obesity and related co-morbidities. As experts on genetics and human biology, Regeneron is excited to join forces with the chemistry and small molecule leaders at AstraZeneca, as we seek to develop new medicines tackling the harmful and costly obesity epidemic.”.

Medicine 52

Medicine Development Research Insulin 52

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Gene Therapy Gene Gene Editing In-Vivo 59

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., Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US. 14, 2020 10:00 UTC.

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Business Development Development Gene Editing In-Vivo 40

pharmaphorum

OCTOBER 8, 2021

The company is also seeking if there is evidence of clonal expansion of the abnormality in in other words passed on to daughter cells – and if it is linked to the gene-editing used to modify the ALLO-501A cells.

Trials 52

Trials Gene Editing Genetics Gene 52

BioSpace

NOVEMBER 17, 2022

Editas Medicine is pausing its ocular gene therapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.

Gene Therapy 42

Gene Therapy Gene Gene Editing Medicine 42

Bioengineer

JULY 23, 2021

“We are creating a platform technology using the organ-on-a-chip approach where tumor biology can be advanced, and new drugs can be identified by recreating the platelet-tumor and platelet-tumor-drug interactions under the influence of flow, supporting blood vessels and the extracellular matrix,” Jain said. ” ###.

Engineer 52

Engineer Gene Editing Clinical Trials Clinical Trials 52

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. That would just slow the whole field down.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Trialfacts

SEPTEMBER 12, 2024

Must not have a history of alcohol or drug abuse within the past 2 years. Must not have been diagnosed with cancer within the past 5 years, excluding completely treated in situ carcinoma of the cervix, and completely treated and resolved non-metastatic squamous or basal cell carcinoma of the skin or melanoma in situ.

Research 52

Research Gene Editing Biobanking Bioinformatics 52

The Pharma Data

NOVEMBER 3, 2020

(Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA).

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Clinical Trials Clinical Trials Gene Editing Genomics 40

Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

pharmaphorum

NOVEMBER 2, 2021

BsAbs also have the potential to improve the delivery of payloads , an isotype or drug, to tumour cells. Payload delivery using monovalent antibodies, such as radioimmunotherapy and antibody-drug conjugates, relies on the direct coupling of a payload to the antibody, which then binds directly at the tumour site for delivery.

Antibody 52

Antibody Immune Response Production Engineer 52

Advarra

NOVEMBER 29, 2022

There have been recent signals indicating the Food and Drug Administration (FDA) plans to provide greater support for CGT, addressing key issues continuing to impede their development. Recent Problems in Cell and Gene Therapy Development. Trend One: Expected Growth in Cell and Gene Therapy IND filings.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

Pharmaceutical Technology

FEBRUARY 13, 2023

Although recently approved drugs can treat seizures more safely, they do not treat the comorbidities that patients experience. Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome.

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Research Gene Gene Therapy Genomics 264