pharmaphorum

JANUARY 29, 2021

“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”. They wanted someone who had lots of experience in drug development, was a molecular biologist, and was stubborn enough to take on CRISPR!” billion in funding.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Delveinsight

JULY 24, 2021

In addition to this, Angelman syndrome pipeline also involves gene therapy candidates such as GTX-101 which is a therapy of GeneTx Biotherapeutics. Another therapy by the same company named GTX-102 is also an antisense therapy; however, it silences gene expression by binding to the RNA molecule that regulates its expression.

Gene Therapy 96

Gene Therapy Gene Gene Expression RNA 96

The Pharma Data

NOVEMBER 8, 2020

BOSTON–( BUSINESS WIRE )– Decibel Therapeutics , a clinical-stage biotechnology company developing novel restorative gene therapeutics to treat hearing loss and balance disorders, today announced the closing of an oversubscribed Series D financing, which raised $82.2M. 9, 2020 11:00 UTC. partner at OrbiMed.

Gene Therapy 40

Gene Therapy Gene Gene Expression Genome 40

XTalks

NOVEMBER 6, 2020

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The gene therapy-based vaccine targets mucosal tissues in the gastrointestinal tract, allowing for greater selectivity and targeted delivery compared to traditional vaccines.

Clinical Trials 75

Clinical Trials Clinical Trials Vaccine Vaccination 75

XTalks

MARCH 13, 2024

Biomarker testing is being pursued to identify biological signs of the disease and provide an approach to develop personalized treatment plans for each patient. As this technology continues to evolve, it is expected to help identify glaucoma in its nascent stages of development.

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

DNA 98

DNA Gene Gene Silencing Genome 98

Roots Analysis

AUGUST 31, 2023

The two types of gene regulation have been discussed below: Positive Gene Regulation: Activator is a protein molecule that helps to initiate a positive gene regulation. When activator binds to the operon, it either speeds up or permits gene expression.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

Pharma Marketing Network

DECEMBER 21, 2020

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. greater safety, transient and reversible effect on gene expression).

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

The Pharma Data

JULY 12, 2021

.” In the new study, Ghosh and colleagues replaced the first and last steps in preclinical drug discovery with two novel approaches developed within the UC San Diego Institute for Network Medicine (iNetMed), which unites several research disciplines to develop new solutions to advance life sciences and technology and enhance human health.

Drugs 52

Drugs Gene Expression Big Data Gene 52

Delveinsight

JANUARY 18, 2021

The first sign of a child suffering from Angelman syndrome is a delay in development, such as the inability to sit without support or making incoherent babbling sounds. Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. GeneTx and Ultragenyx are co-developing the therapy.

Marketing 52

Marketing Gene Drugs Protein 52

The Pharma Data

OCTOBER 15, 2020

“For cell therapy products to mature into ‘real’ products that deliver on the promises of 10 years ago, they must be scalable – which drives affordability – and they must solve their purity issues.”. One of the challenges is balancing the clinical and manufacturing aspects of development. “If It’s not a quick fix,” he said.

Manufacturing 52

Manufacturing Gene Therapy Gene Gene Expression 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA 52

DNA Gene Gene Silencing Genome 52

The Pharma Data

DECEMBER 27, 2020

The drug is a humanized IgG4 monoclonal antibody that blocks CCR5 and is being developed for COVID-19, HIV and metastatic triple-negative breast cancer. TSHA-101 is an investigational gene therapy designed to deliver the HEXA and HEXB genes driven by a single promoter within the same AAV9 construct, dubbed a bicistronic vector.

Trials 52

Trials Antibody Gene Therapy Gene 52

Roots Analysis

JANUARY 14, 2024

With the increased interest and gradual shift of investment from small molecule drugs to biologics and the establishment of several biologics manufacturing companies / biologics CMOs, more than 250 biologic therapies and vaccines have been developed, globally.

Contract Manufacturing 40

Contract Manufacturing Protein Manufacturing Production 40

XTalks

DECEMBER 24, 2020

Focusing on biologics, vaccine development and policy platforms in anticipation of an eventual outbreak is why we’re where we are today in terms of the positive side [of the pandemic].”. Most antibody drugs and vaccines have been developed to target parts of the spike protein. Pandemic Efforts.

Life Science 111

Life Science Vaccination Vaccine Gene 111