XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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Pharmaceutical Technology

AUGUST 17, 2022

The rights to the oRNA-LNP technology platform of Orna will be retained by the company, which will also progress various other fully owned programmes in oncology and genetic disease areas. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.

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pharmaphorum

OCTOBER 26, 2020

Bayer is making clear its ambitions in cell and gene therapy with a $4 billion acquisition of US biotech Asklepios BioPharmaceutical, also known as AskBio. AskBio’s portfolio includes pre-clinical and clinical stage candidates for the treatment of neuromuscular, central nervous system, cardiovascular and metabolic diseases.

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pharmaphorum

JANUARY 11, 2021

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO. The post bluebird bio to split into oncology and gene therapy specialists appeared first on. million price tag.

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Pharmaceutical Technology

MARCH 1, 2023

Vertex will also handle all subsequent development, manufacturing, and commercialisation activities. Tevard CEO and co-founder Daniel Fischer said: “Given Vertex’s proven track record of developing novel therapies to treat serious disease, we’re thrilled to partner with them on this important effort. “By

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Pharmaceutical Technology

JANUARY 12, 2023

Under the research partnership deal, the companies will develop new precision genetic medicines for a severe form of genetic dilated cardiomyopathy (DCM). Our pipeline of programmes focused on neuromuscular and cardiac rare disease gives us deep insight into the diverse and complex nature of dilated cardiomyopathy.”.

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XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Marson’s group initially collaborated with CRISPR pioneer Jennifer Doudna, PhD, in 2015.

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Delveinsight

FEBRUARY 11, 2021

Avrobio gene therapy eradicates toxic substrate in Fabry patient. The first Fabry disease patient treated with Avrobio’s Plato gene therapy platform in a phase 2 trial has undergone the complete clearance of toxic kidney substrate. Median overall survival in those patients was 5.8

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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.

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Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. The evolution of NGS technologies has made it possible to sequence thousands of genes with a suspected genetic disease predisposition, in a very short period of time and at minimal costs.

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XTalks

NOVEMBER 27, 2023

Pompe disease, also referred to as acid-maltase disease and glycogen storage disease II, is an uncommon genetic disorder characterized by the gradual weakening of both cardiac and skeletal muscles. The body relies on this enzyme to break down glycogen, a stored form of sugar utilized for energy production.

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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.

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The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

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XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. The disease usually presents within the first year of life, displaying subtle signs that can take years to develop. Due to this, the disease can be misdiagnosed or not even recognized for a while.

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The Pharma Data

DECEMBER 9, 2020

Rocket intends to use the net proceeds from this offering to further fund the development of its pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the United States and Europe, accelerating the buildout of in-house manufacturing capabilities, and for general corporate purposes.

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Roots Analysis

AUGUST 12, 2024

Hybridization Tests – These tests utilize complementary DNA or RNA probes to detect and quantify specific genetic targets, enabling the identification of pathogens, genetic variations, and gene expression patterns. Such test can be used for gene expression profiling, genotyping and detecting chromosomal abnormalities.

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The Pharma Data

DECEMBER 17, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (Paris:LYS) (FR0013233475 – LYS), a phase 3 gene therapy platform Company targeting central nervous system (CNS) diseases, announces a change in the governance and control of KGA, a company co-owned by Karen Aiach and which currently owns approximately 6% of Lysogene’s capital.

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The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869).

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The Pharma Data

OCTOBER 18, 2020

Telomeres degrade and shorten with age and can become excessively damaged in certain genetic diseases, as well as from lifestyle factors such as smoking, poor diet, and chronic stress. Likewise, these scientists produced “telomere-depleted” mice by inactivating a gene for telomerase, the enzyme that extends telomeres.

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The Pharma Data

NOVEMBER 2, 2020

This condition is caused by a mutation in the fibroblast growth factor receptor 3 gene ( FGFR3 ), a negative regulator of bone growth. More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. In the U.S., About BioMarin.

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XTalks

APRIL 24, 2023

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

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XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

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pharmaphorum

OCTOBER 25, 2022

Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biotech Taysha demonstrates. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.

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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.

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XTalks

MARCH 21, 2024

The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

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XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. 1. Cell and Gene Therapies. In 2023, a number of gene therapies are expected to get the FDA green light.

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STAT News

FEBRUARY 22, 2023

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.

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The Pharma Data

AUGUST 3, 2021

SVP, Chief Medical Officer, Novartis Gene Therapies. “We Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA. This route of administration has the potential to open up access for older patients to all the benefits of gene therapy.

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The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

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The Pharma Data

DECEMBER 20, 2020

In the rejection, the agency indicated they need to conduct pre-approval inspections at two of scPharmaceuticals’ third-party manufacturing facilities that they couldn’t do because of travel restrictions related to the COVID-19 pandemic. It is caused by a defective and/or missing CFTR protein caused by mutations in the CFTR gene.

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The Pharma Data

NOVEMBER 8, 2020

The New Drug Application (NDA) for the drug is based on data from four Phase III trials in pediatric patient populations from the age of 6 to 17 years, two Phase II trials, several Phase I trials, a long-term open label extension study, preclinical testing, and drug manufacturing data. Adamis Pharmaceuticals’ Zimhi for Opioid Overdose.

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The Pharma Data

OCTOBER 15, 2020

Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. 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. Additionally, Parag V.

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The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. About Primary Hyperoxaluria Type 1 (PH1) PH1 is an ultra-rare genetic disease that affects an estimated one to three individuals per million in the United States and Europe. Oxlumo should be administered by a healthcare professional.

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XTalks

DECEMBER 13, 2023

The year 2022 has proven to be a momentous period for Fulgent Genetics, marked by significant expansion of its product portfolio across various medical conditions. At the heart of this platform lies a proprietary technology that pioneers a novel method for manufacturing synthetic DNA by “writing” DNA on a silicon chip.

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XTalks

DECEMBER 22, 2021

Formulas can be generated in days and clinical-grade manufacturing can be accomplished in weeks. The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. They can be developed and adapted quickly. The deal is worth $1.5

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Delveinsight

JANUARY 12, 2021

BeiGene has announced the collaboration with Novartis Pharma AG to develop, manufacture, and commercialize BeiGene’s anti-PD-1 antibody tislelizumab in the United States, Canada, Mexico, member countries of the European Union, United Kingdom, Norway, Switzerland, Iceland, Liechtenstein, Russia, and Japan. and Europe.

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