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

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.

Genome 40

Genome Genomics Gene In-Vivo 40

BioTech 365

OCTOBER 12, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the successful completion of a RMB 450 million (approximately USD 67 million) Series … Continue reading → BEIJING & CAMBRIDGE, Mass.–(BUSINESS

Gene Editing 40

Gene Editing Genome Genomics Gene 40

Delveinsight

SEPTEMBER 14, 2021

ADARx Pharmaceuticals, a biotechnology company developing RNA targeting therapeutics , announced the completion of a USD 75 million Series B financing to progress its drug development pipeline. Vor Biopharma’s AML drug secures Fast Track Designation. ADARx bags USD 75 Million to advance its RNA tech pipeline. Carl Gordon, Ph.D.,

RNA 67

RNA Clinical Trials Clinical Trials Drugs 67

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

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

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.

Business Development 40

Business Development Development Gene Editing In-Vivo 40

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.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

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

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. The potential for CRISPR gene editing to offer a therapeutic option to patients is clear, based on its mechanism, but there are already gene editing therapeutics on the market.

Gene Editing 52

Gene Editing DNA Gene Genetics 52

The Pharma Data

JANUARY 24, 2021

“We are excited to welcome Chris, a highly regarded drug developer and team builder who brings to Notch great depth of experience and expertise in development of cell therapies, gene editing, and cell engineering spanning discovery through IND,” said David Main , President and Chief Executive Officer of Notch.

Engineer 52

Engineer Gene Editing Genomics Genome 52

Delveinsight

AUGUST 27, 2020

Freenome’s diagnostic searches for direct indication of tumors and signs of the immune system’s response, integrating genomics, transcriptomics, methylomics, and proteomics technologies. Targeted drug combo attacks breast cancer brain metastases in mice. White fat, in contrast, can build up and cause obesity.

Vaccination 53

Vaccination Vaccine Research Trials 53

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”.

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

XTalks

MAY 12, 2021

Dubbed “CRISPRoff,” the technology targets the epigenome to silence genes involved in diseases, with applications in cancer, AML and other conditions with a heritable component. Read the full articles here: Epigenetic Editing with CRISPR Might Be Easier Than We Thought. New Study Looks at Effects of ADHD Drugs in Preschoolers.

Life Science 40

Life Science Gene Expression Gene DNA 40

The Pharma Data

NOVEMBER 3, 2020

Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process. About LogicBio Therapeutics.

Clinical Trials 40

Clinical Trials Clinical Trials Gene Editing Genomics 40

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.

Research 264

Research Gene Gene Therapy Genomics 264

pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. Its main platform – called X-editing – can generate CRISPR drugs with improved activity, specificity and deliverability compared to rivals, according to the start-up’s website.

Gene Editing 69

Gene Editing Gene In-Vivo Clinical Trials 69

Cloudbyz

MARCH 6, 2024

Traditionally, pharmaceutical therapies have primarily consisted of small molecule drugs, commonly known as pills. This shift signifies not just a change in the way drugs are manufactured and administered, but also a fundamental transformation in the approach to treating diseases at their root cause.

Gene 99

Gene Immune Response Manufacturing Gene Therapy 99

The Pharma Data

JANUARY 10, 2021

Nasdaq: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. Enrolled patients will receive a single administration of EDIT-301.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

XTalks

NOVEMBER 30, 2023

In 2022, the US Food and Drug Administration (FDA) granted approval to the medication Sunlenca (lenacapavir) for adults living with multi-drug resistant HIV-1 infection. Developed by Gilead, Sunlenca is the first drug in a novel class known as capsid inhibitors to receive FDA approval for treating HIV-1.

FDA Approval 111

FDA Approval Gene Therapy Gene Clinical Trials 111

The Pharma Data

JANUARY 12, 2021

“There is an incredible unmet need in some diseases that can only be addressed through cell and gene therapies.”. As a result, the company settled on four areas of focus – iPSC, allogenic CAR-T, genome mutations and gene editing. However, she said the company wants to strengthen its position in gene editing.

Gene Therapy 52

Gene Therapy Gene Gene Editing Business Development 52

XTalks

MARCH 28, 2024

Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment. For example, gene editing can possibly be used to correct cancer-causing mutations or enhance the effectiveness of chimeric antigen receptor (CAR) T-cell therapy.

Gene Editing 59

Gene Editing Life Science Clinical Trials Clinical Trials 59

Delveinsight

FEBRUARY 25, 2021

Beam adds to drug delivery stable with USD 120 Million GuideTx buy. The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. If they eliminated the T cells, the therapy stopped working.

DNA 52

DNA In-Vivo Genetics Medicine 52

The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., LNPs are spheric drug delivery bodies which can be equipped with therapeutic payloads for intracellular delivery. Financial details were not disclosed. Source link: [link]

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

pharmaphorum

FEBRUARY 3, 2021

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

Gene Editing 98

Gene Editing Gene DNA Engineer 98

Pharmaceutical Technology

FEBRUARY 20, 2023

On the other hand, a lack of diversity in clinical trial populations, particularly when the disease population is significantly underrepresented, greatly diminishes the quality of data obtained for drug safety and efficacy profiles. This may sound obvious, but it is still a major hurdle in drug development.

Research 130

Research Clinical Trials Clinical Trials Trials 130

Pharma Marketing Network

JANUARY 20, 2021

Because COVID-19 can progress rapidly from a respiratory disease to a multisystemic inflammatory and coagulopathic disorder, there is a need to develop not only antiviral therapies, but also drugs that block complement activation, coagulation, and cytokines, according to Prabhavathi Fernandes (Chair, National Biodefense Science Board).

Vaccination 52

Vaccination Vaccine In-Vivo Genetics 52

The Pharma Data

JANUARY 24, 2021

The drug appears to inhibit IL-6, IL-1beta, TNF-alpha and other pro-inflammatory cytokines and chemokines. The drug did not differ statistically from placebo at 48 weeks on the prespecified co-primary endpoints on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 and the Clinical Dementia Rating Scale Sum of Boxes.

Trials 52

Trials Allergies In-Vitro Antibody 52

The Pharma Data

DECEMBER 2, 2020

Food and Drug Administration (FDA) for Onivyde (irinotecan liposome injection) for study patients with small cell lung cancer (SCLC) who progressed following a first-line platinum-based regimen. The drug is a small-molecule inhibitor of the S. Paris-based Ipsen secured Fast Track designation from the U.S. in Mainland China.

Drugs 52

Drugs Gene Editing Genomics Genome 52

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. Drugs, Testing and Vaccines. This makes the latter potentially easier to target with drugs and vaccines.

Life Science 111

Life Science Vaccination Vaccine Gene 111

XTalks

JANUARY 6, 2025

This blog highlights the top 10 biotech trends for 2025, showcasing cutting-edge technologies and approaches that promise to redefine patient care, accelerate drug development and expand our understanding of human biology. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming genetic diseases in the near future.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111