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).

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

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

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

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

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.

Gene Splicing 130

Gene Splicing Gene DNA Recombinant DNA Technology 130

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

JANUARY 17, 2021

But that number doesn’t quite reflect the company’s likely market valuation , which is expected to be between $9 billion and $12 billion. 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.

In-Vivo 52

In-Vivo Marketing Engineer Genetics 52

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

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 Vaccination Vaccine 245

Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. The present Angelman syndrome treatment market revolves around the management of symptoms rather than curing the condition. . Besides underdiagnosis, a lack of standardized treatment option forms a huge clinical unmet need.

Marketing 52

Marketing Gene Drugs Protein 52

Roots Analysis

MARCH 1, 2023

Genotoxicity testing refers to the evaluation of detrimental effects of chemical or physical agents on the genetic processes and related hereditary material of living cells. Mechanism of Genotoxicity / Mutagenicity The interaction of genotoxins / mutagens with the structure of DNA causes damage to the genetic material.

Genotoxicity 52

Genotoxicity DNA Genetics Bacteria 52

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

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. 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 Contamination Production Gene Therapy 40

pharmaphorum

APRIL 12, 2021

C4XD said it has developed a molecule small enough to be formulated into a pill, which has also been able to block IL-17 in vivo. This follows a tie-up between C4XD, Sano Genetics and BenevolentAI to accelerate research into inflammatory bowel disease, which was signed in February.

Drugs 52

Drugs In-Vivo Licensing Licensing 52

pharmaphorum

AUGUST 24, 2021

It will focus on ‘ex vivo’ therapies, which involves harvesting cells from patients, modifying them and then infusing then back into the body. “This new collaboration further expands our toolkit in cell and genetic therapies,” said Bastiano Sanna, Vertex’ chief of cell and genetic therapies. .

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 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

XTalks

APRIL 24, 2023

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

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. 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

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

Delveinsight

JANUARY 12, 2021

The companies believe that their candidate in its original IgG format has shown potent neutralization activity in in vitro assays and in an in vivo animal model. Already in markets in China, BeiGene’s Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fc? and Europe.

Licensing 52

Licensing Licensing Antibody Gene Therapy 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. This alone makes them much safer than most conventional vaccines on the market today.

Protein 40

Protein Vaccine Vaccination Immune Response 40

The Pharma Data

OCTOBER 15, 2020

marketing and diagnostics. 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. The Advance awards recognize the work of remarkable global Australians making an extraordinary impact worldwide.

In-Vivo 52

In-Vivo Business Development Manufacturing Development 52

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

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

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

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. 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.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Roots Analysis

JANUARY 14, 2024

Notably, biologics have higher success rates than conventional small-molecule drugs owing to lesser off-target toxicity of biologics, which is one of the key reasons of failures in small molecule drugs development. They are different from small molecules in terms of their size and complexity.

Contract Manufacturing 40

Contract Manufacturing Protein Manufacturing Production 40

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

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

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