Scienmag

OCTOBER 14, 2020

Known as myofibrillar myopathies, these rare genetic diseases lead to progressive muscle wasting, affecting muscle function and causing weakness.

Genetic Disease 67

Genetic Disease Genetics Protein Research 67

XTalks

MAY 26, 2023

Vyjuvek is also the first drug approved to treat the disease and is Krystal’s first approved product. DEB is a genetic disorder characterized by very fragile skin that rips and blisters easily even from minor friction (like rubbing or scratching) or injury, resulting in open wounds that are prone to skin infections and fibrosis.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . A rare inherited neurodegenerative disease, it’s thought to affect 62,000 people in the European Union.

Gene Therapy 98

Gene Therapy Gene Trials Clinical Trials 98

Drug Discovery World

AUGUST 28, 2023

It is the method for removing unwanted debris, e.g., old proteins, and breaking them down so their constituent parts can be used again. The ones we have focussed on, because the data is so promising and the opportunity so big, are neurodegenerative disorders, in particular Parkinson’s disease and Amyotrophic Lateral Sclerosis (ALS).

Genetics 52

Genetics Protein Genetic Disease Clinical Trials 52

Pharma Mirror

APRIL 21, 2024

With the rapid development of biotechnology and molecular medicine, the introduction of mRNA as a vaccine or therapeutic agent enables the production of almost any desired functional protein/peptide within the human body.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

XTalks

AUGUST 6, 2020

This label expansion, including the expansion of the age range in all approved indications, further demonstrates that the FDA process can continue to enable broader patient access to appropriately tested regulatory approved cannabinoid medicines. TSC is a rare genetic disease that affects approximately 1 in 6,000 people.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

The Pharma Data

MARCH 16, 2021

in patients with VHL disease-associated RCC. Von Hippel-Lindau disease is a rare genetic condition for which there is no systemic treatment option available and is associated with a high risk of cancer development in multiple organs. Proteins known as hypoxia-inducible factors, including HIF-2?, About Belzutifan.

Drugs 52

Drugs Trials Genetics Protein 52

Delveinsight

AUGUST 26, 2021

Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease. Shape’s RNA editing technologies can modify the RNA sequence, which makes the body’s protein building blocks.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 87

Genome Genomics Genetics Genome Project 87

Drug Discovery World

NOVEMBER 14, 2023

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP).

FDA Approval 52

FDA Approval Gene Clinical Trials Clinical Trials 52

The Pharma Data

OCTOBER 27, 2020

The company plans to tackle the existing challenges of precision medicine with a three-prong approach – developing better medicines for known cancer-driving mutations, developing drugs for targets previously thought to be undruggable, and finding new, untapped targets that could lead to even better drugs. Sirnaomics. Be Biopharma .

Gene 52

Gene Gene Therapy Medicine Genetics 52

Drug Discovery World

MAY 11, 2023

However, she also presented thoughts in a technical update, Targeted Protein Degradation – Progress, Direction and Prospects. Last, but certainly not least, Samsara Therapeutics is a biopharma company aiming to discovery first-in-class therapeutics that treat rare genetic and neurodegenerative disease, and extend healthy lifespan.

Life Science 52

Life Science Genetics Vaccination Vaccine 52

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 76

Genome Genomics Genetics Genome Project 76

The Pharma Data

DECEMBER 8, 2020

Through its Shielded Living Therapeutics platform, the company is developing functional cures for chronic diseases. The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. .

RNA 52

RNA Antibody Life Science Protein 52

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

DECEMBER 13, 2023

These efforts encompass programs related to the shingles vaccine, Tdap vaccine and plague vaccine, reinforcing Dynavax’s commitment to advancing the field of vaccines and medicine. 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.

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Genetics Vaccination Vaccine DNA 111

The Pharma Data

DECEMBER 20, 2020

Elizabeth’s Medical Center and associate professor of Urology at Tufts University School of Medicine, said at the time, “The results of the EMPOUR study over the 52-week period demonstrated the sustained benefits of fibegron. It is caused by a defective and/or missing CFTR protein caused by mutations in the CFTR gene.

In-Vitro 52

In-Vitro FDA Approval Manufacturing Drugs 52

Drug Discovery World

FEBRUARY 28, 2023

As we turn our focus to new potential applications and disease areas for the platform, scientists and companies must consider the potential life-saving impact on rare, inherited diseases, says Archana Gupta , PhD, staff scientist in genetic sciences at Thermo Fisher Scientific.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52

The Pharma Data

DECEMBER 3, 2020

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease.

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

Drug Discovery World

MARCH 6, 2024

The development of effective new disease-modifying treatments in neurodegenerative and neuropsychiatric disorders has been hindered by their inherent genetic complexity, environmental influences, and clinical variability. This is comparatively easier to accomplish in these diseases because they are relatively monogenic.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

Drug Discovery World

OCTOBER 12, 2023

She says: “From turning genes off to look at the effect on cellular function, making single base changes within a coding or non-coding region to see if it impacts protein function, to testing drug candidates on genetic variants, we now have unprecedented genetic control to understand the link between genetics, disease and treatment.”

Drugs 59

Drugs Life Science Genome Genomics 59

The Pharma Data

JULY 6, 2021

Haemophilia A is a serious, inherited bleeding disorder in which a person’s blood doesn’t clot properly, as they either lack or do not have enough of a clotting protein called factor VIII. 11 People with haemophilia A either lack or do not have enough of a clotting protein called factor VIII.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

pharmaphorum

JANUARY 10, 2022

The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases. Similar to Pfizer, Sanofi noted that bringing the companies into its portfolio would allow it to leverage research capabilities in oncology and inflammatory disease.

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RNA Vaccination Vaccine Pharma Companies 109

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. On November 19, the European Commission granted marketing authorization for Oxlumo for the treatment of PH1 in all age groups, following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). President of R&D at Alnylam.

FDA Approval 52

FDA Approval Production RNA Medicine 52

XTalks

DECEMBER 22, 2021

RNA, and its protein-generating form messenger RNA (mRNA) discovered in 1961 , has quickly transitioned from being an obscure, finicky molecule that is difficult to work with, to becoming a significant cornerstone of therapeutic innovation in pharma and biotech. The RNA Revolution: From mRNA Vaccines to RNA Editing. RNA Therapeutics.

Life Science 98

Life Science RNA Vaccination Vaccine 98

The Pharma Data

AUGUST 3, 2021

The use of a sham procedure in STEER is included to provide a comparison group for an unbiased collection and assessment of efficacy, safety and tolerability of OAV-101 IT for this older population where the disease progression is slower. Spinal Muscular Atrophy (SMA) ?

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

JANUARY 9, 2021

Boxer, MD Research Professor of Pediatrics and Communicable Diseases Department of Pathology Michigan Medicine at the University of Michigan and investigator in the Phase 3 study. Additionally, the European Medicines Agency (EMA) requested one-year results from the full Phase 3 study to inform their benefit-risk assessment.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Drug Discovery World

FEBRUARY 22, 2024

With the breadth and range of central nervous system disorders – for example Parkinson’s disease and multiple sclerosis – facing the global population there is an unmet need within this area of drug discovery. They focused on miRNAs, which regulate multiple target proteins controlling the cellular processes in the amygdala.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

The Pharma Data

SEPTEMBER 22, 2020

All patients deserve a gene therapy designed to address the genetic root cause of their disease with a single dose. Zolgensma was approved by the US Food and Drug Administration in May 2019 and represents the first approved therapeutic in the company’s proprietary platform to treat rare, monogenic diseases using gene therapy.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52