XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform. Related: Red Meat Allergy Test Gets FDA Clearance. “We

Clinical Trials 75

Clinical Trials Clinical Trials Vaccine Vaccination 75

Pharmaceutical Technology

MARCH 1, 2023

Nedisertib is under clinical development by Merck and currently in Phase I for Neuroendocrine Tumors. The drug candidate targets DNA-dependent protein kinase (DNA-PK). It was also under development for small cell lung cancer, rectal cancer and chronic lymphocytic leukemia (CLL). It is a new chemical entity.

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

Pharmaceutical Technology

MARCH 1, 2023

Nedisertib is under clinical development by Merck and currently in Phase I for Head And Neck Cancer Squamous Cell Carcinoma. The drug candidate targets DNA-dependent protein kinase (DNA-PK). It was also under development for small cell lung cancer, rectal cancer and chronic lymphocytic leukemia (CLL).

Gene Editing 100

Gene Editing DNA Cosmetics Life Science 100

pharmaphorum

JULY 5, 2022

The new partnership will apply Mogrify’s cellular reprogramming and bioinformatics expertise to try to identify transcription factors – proteins which can turn specific genes on or off – that can be used to generate new cochlear hair cells. An estimated 1.57

In-Vivo 110

In-Vivo Bioinformatics Genetics Licensing 110

pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

The Pharma Data

JANUARY 27, 2021

Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBFT02, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of patients with Frontotemporal Dementia (FTD) with granulin (GRN) mutations. PGRN is a complex and highly conserved protein.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

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

JULY 18, 2022

There have recently, however, been some notable developments towards improving the care of bronchiectasis patients. These include the ongoing clinical development of AstraZeneca’s Fasenra (benralizumab), Insmed’s brensocatib and Novartis’ icenticaftor (QBW251).

Marketing 147

Marketing Trials Development Protein 147

pharmaphorum

SEPTEMBER 12, 2022

Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Cancer cells and tumours behave differently than normal cells and have changes in their genes that make them different from an individual’s normal cells.

Genomics 105

Genomics Genome Clinical Trials Clinical Trials 105

The Pharma Data

DECEMBER 13, 2020

2] Pre-clinical studies have shown that these differentiated properties of HMBD-001 lead to robust and sustained tumor growth inhibition in multiple HER3 cancer models, including those with NRG1-fusions. Our highly experienced teams in Singapore and the US span antibody discovery, pharmacology, production and clinical development.

Clinical Development 40

Clinical Development Medicine Development Antibody 40

pharmaphorum

AUGUST 10, 2020

for a single shot of the gene therapy, making it the most expensive drug in the world by many people’s reckoning, although the manufacturer argues this is justified given the costs of treating the condition in later life. Evrysdi is designed to treat SMA by increasing production of the survival of the motor neuron (SMN) protein.

FDA Approval 59

FDA Approval Drugs Gene Therapy Protein 59

The Pharma Data

DECEMBER 31, 2020

where he led global clinical development programs including clinical strategy, clinical development, and regulatory affairs. Prior to Taiho, he held the positions of senior vice president of Clinical Oncology and CMO at Geron Corporation. and CMO for Taiho Oncology, Inc.

Gene Therapy 52

Gene Therapy Engineer Gene Research Analyst 52

The Pharma Data

MARCH 23, 2021

Vitrakvi is a highly selective TRK inhibitor exclusively designed to treat tumors that have an NTRK gene fusion. TRK fusion cancer occurs when an NTRK gene fuses with another unrelated gene, producing an altered TRK protein. About Vitrakvi (Larotrectinib). About Oncology at Bayer.

Genomics 52

Genomics Genome Gene Protein 52

pharmaphorum

NOVEMBER 18, 2021

Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 Once in the blood, the GalXC molecules travel to the liver, where they enter hepatocyte cells and can switch protein production on or off.

Gene Silencing 52

Gene Silencing RNA Protein Gene 52

pharmaphorum

OCTOBER 13, 2022

million in future payments tied to preclinical, clinical development, and commercial achievements. Vividion will contribute its ability to find previously unknown functional binding pockets on molecules like proteins or transcription factors that can be targeted with drug compounds. Vividion is making an upfront payment of $17.5

Gene Therapy 64

Gene Therapy Genomics Genome Drugs 64

The Pharma Data

DECEMBER 10, 2020

executive vice president of research and development. “We We look forward to working closely with the FDA throughout the clinical development process to bring this potential new innovative treatment to patients as quickly as possible.”. Safe Harbor Statement.

Containment 52

Containment Clinical Development Development Protein 52

The Pharma Data

JULY 6, 2021

Enrolment is currently underway for a pivotal Phase 3 trial for AL001 in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). billion in clinical development, regulatory and commercial launch-related milestone payments. This population represents 5% to 10% of all people with FTD.

Genetics 52

Genetics Gene Antibody Development 52

The Pharma Data

OCTOBER 27, 2020

The Massachusetts-based biotech is investing in their proprietary “Gene Traffic Control” system, which tells the cells when to express which genes where and in what order within the chromatin regulatory system. Be Bio is a leader in developing B cells as medicines. The company currently has over 10 programs in its pipeline.

Gene 52

Gene Gene Therapy Medicine Development 52

The Pharma Data

MAY 31, 2022

Of the babies with 2 or 3 copies of the SMN2 gene (n=6), 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% of infants could walk independently. More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings.

FDA Approval 52

FDA Approval Protein In-Vitro Medicine 52

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

The Pharma Data

APRIL 29, 2022

Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. About Evrysdi® (risdiplam) Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency.

Protein 52

Protein In-Vitro Medicine Development 52

pharmaphorum

APRIL 15, 2021

There are several treatment options now available for SMA, with Novartis’ ultra-pricey Zolgensma offering a one-off gene therapy, while Biogen’s antisense drug Spinraza instructs the body to produce a working version of the SMN protein that is the root cause of the disease.

Gene Therapy 52

Gene Therapy Protein Clinical Development Gene 52

Delveinsight

AUGUST 20, 2020

FDA rejects BioMarin’s gene therapy and Gilead’s filgotinib over concerns. The FDA refused to approve BioMarin’s hemophilia A gene therapy valoctocogene roxaparvovec. Amyloid oligomers are clusters of individual amyloid proteins and represent a target that drugs can hit before full-blown amyloid plaques form.

Gene Therapy 52

Gene Therapy Research Gene Antibody 52

Delveinsight

DECEMBER 24, 2020

GPR35 is a G protein-coupled receptor (GPCR) that was recognized in the late 1990s. Information on the function of GPR35 was slow to develop because of the issues, including an initial lack of ligands, which regulate the receptor, and the inability to study its function in rodent models.

Research 52

Research Trials Clinical Trials Clinical Trials 52

XTalks

JUNE 9, 2022

When a child born from parents with FH genes, inherits one FH gene from each parent, they might develop homozygous familial hypercholesterolemia (HoFH). Children suffering from HoFH develop xanthanomas within the first few years of their life. The ANGPTL3 protein inhibits the enzyme activity of lipoprotein lipase (LPL).

Trials 52

Trials Antibody Genetics Gene 52

The Pharma Data

MARCH 11, 2021

Preclinical studies are ongoing and will continue over the next several months to evaluate whether MRT5500, as well as additional mRNA vaccine candidates, will induce neutralizing antibodies against the emerging SARS-CoV-2 variants, with the potential to inform current and future clinical development. About Translate Bio.

Clinical Trials 52

Clinical Trials Clinical Trials Vaccine Vaccination 52

Delveinsight

JULY 30, 2020

Adrenoleukodystrophy (ALD/ X-ALD) is a rare, X-linked disorder secondary to a mutation in the ABCD1 gene, which encodes for a peroxisomal membrane protein, resulting in an excessive accumulation of very-long-chain fatty acids (VLCFAs) in the brain and adrenal glands. Adrenoleukodystrophy Pipeline.

Hormones 52

Hormones Marketing Gene Trials 52

The Pharma Data

OCTOBER 14, 2020

About VBL’s VB-600 Platform VBL is conducting two parallel drug development programs that are exploring the potential of MOSPD2 (motile sperm domain-containing protein 2), a protein that VBL has identified as a key regulator of cell motility, as a therapeutic target for inflammatory diseases and cancer.

Antibody 40

Antibody Gene Therapy Containment Clinical Trials 40

Roots Analysis

OCTOBER 30, 2023

These RNA based therapeutics play a crucial role in protein production and regulation of gene functions. Self-amplifying RNA (saRNA): Self amplifying RNA is an engineered form of RNA, combining the amplifying genome (native to certain viruses) to the therapeutic gene of interest.

RNA 40

RNA Vaccine Vaccination Development 40

The Pharma Data

JANUARY 20, 2021

” “Dynacure is a leader in rare disease drug development, as evidenced by its lead product candidate DYN101, which is in clinical development for the treatment of Myotubular and Centronuclear Myopathies for which there are no FDA or EMA approved therapeutics,” said Ms. ” Ms.

Life Science 40

Life Science Development Genetics Production 40

The Pharma Data

JANUARY 3, 2021

The Phase 1/2 clinical trial will primarily investigate the safety and tolerability of INZ-701 and characterize its pharmacokinetic and pharmacodynamic profile, including plasma pyrophosphate (PPi) and other biomarker levels, to establish a recommended dosing regimen for further clinical development. About INZ-701.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Containment 52

The Pharma Data

NOVEMBER 2, 2020

Past research has suggested that people who carry certain variants in the FGF21 gene tend to have a sweet tooth and a preference for carbohydrates. But the protein is cleared from the body too quickly to be useful, explained Arora, who was with biotech company Genentech at the time of the study. More information. 2, 2020, online.

Antibody 52

Antibody Drugs Hormones Insulin 52

The Pharma Data

OCTOBER 19, 2020

Caris Precision Oncology Alliance members also have access to the Caris Pharmatech oncology trial network, which can help reduce the time it takes to identify and connect appropriate patients with novel targeted cancer therapies in clinical development.

Life Science 52

Life Science Clinical Trials Clinical Trials RNA 52

XTalks

MAY 9, 2024

Second, new modalities, such as antibody-drug conjugates, bispecific antibodies and cell and gene therapies, have shown great promise, offering potential in previously difficult-to-treat diseases, including advanced cancer and rare diseases.

Antibody 52

Antibody Clinical Trials Clinical Trials Medicine 52

The Pharma Data

OCTOBER 28, 2020

Five patients between the ages of 5 and 15 with deletions in the maternal UBE3A gene region were enrolled in the first three cohorts and are included in the interim data analysis. Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. About Angelman Syndrome.

Protein 40

Protein Production Development Drugs 40

The Pharma Data

JANUARY 13, 2021

Food and Drug Administration (FDA) has designated as a Fast Track development program the investigation of Brilacidin as a potential treatment for COVID-19. Brilacidin is a first-in-class Host Defense Protein (HDP) mimetic with antiviral, anti-inflammatory and antibacterial properties.

Clinical Trials 52

Clinical Trials Clinical Trials Trials In-Vitro 52

The Pharma Data

JUNE 26, 2021

The safety profile of PDS in the clinical trial setting is well understood and will continue to be closely monitored. Roche has a robust phase III clinical development programme underway for PDS, including the Portal, Pagoda and Pavilion studies. 9 Both the Pagoda and Pavilion trials are actively recruiting participants.

Drug Delivery 52

Drug Delivery Clinical Trials Clinical Trials Medicine 52