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

DEB patients are also at an increased risk of aggressive skin cancer.DEB usually presents at birth and is caused by one or more mutations in the COL7A1 gene, which encodes type VII collagen (COL7), an important protein that helps connect, strengthen and stabilize the outer and middle layers of the skin.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

pharmaphorum

JULY 20, 2022

The Cambridge, Massachusetts biotech has discovered that areas of the non-coding parts of the human genome – referred to by its chief executive Josh Mandel-Brehm (pictured above) as the “dark side” of the genome – actually produce regRNAs that control the expression of the 2% that codes for proteins.

RNA 52

RNA Genetic Disease Drugs Protein 52

The Pharma Data

DECEMBER 3, 2020

Initial results from the study show significantly diminished neurocognitive abilities in children with Dravet syndrome such that 10 year-olds are functioning below the level of a healthy 1 year-old even while they are being treated with available anti-epileptic medicines. STK-001 is designed to upregulate Na V 1.1

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 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

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

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. is a late-stage oncology company dedicated to developing targeted therapeutics for the treatment of metastatic disease. million and $38.8 million and $33.9

Genetics 111

Genetics Vaccination Vaccine DNA 111

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.

RNA 111

RNA Vaccine Vaccination Pharma Companies 111

The Pharma Data

MARCH 16, 2021

Additionally, this study evaluated response rates in other common VHL disease-associated tumors, including pancreatic cysts, pancreatic neuroendocrine tumors, central nervous system (CNS) hemangioblastomas, and retinal hemangioblastomas. Proteins known as hypoxia-inducible factors, including HIF-2?, About Belzutifan.

Drugs 52

Drugs Trials Genetics Protein 52

pharmaphorum

SEPTEMBER 9, 2020

At the time, the Sanger Institute was working on the Deciphering Developmental Disorders study (DDD), which looked at the approximately 1-2% of the genome that is responsible for making proteins in patients with rare diseases. It’s essential to have hope in the future and in the promise that genomic medicine can bring.”.

Genome 101

Genome Genomics Genetics Genome Project 101

XTalks

OCTOBER 8, 2024

Apitegromab is a monoclonal antibody that selectively binds to and inhibits myostatin, a protein that limits muscle growth. Current therapies mainly address motor neuron loss, leaving a need for treatments that directly target muscle weakness.

Trials 52

Trials Antibody Genetics Medicine 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 Development 52

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

XTalks

JANUARY 3, 2025

Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism. People with FCS often have triglyceride levels higher than 880 mg/dL, compared to a healthy target level of below 150 mg/dL. FCS also severely impacts quality of life, causing chronic fatigue and recurrent stomach pain.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

XTalks

SEPTEMBER 16, 2024

HAE is caused by a deficiency or dysfunction of the C1-inhibitor, a protein involved in regulating inflammation. HAE affects approximately one in 50,000 people globally, and there is currently no cure for the disease. These attacks can be unpredictable, often leading to life-threatening situations when they affect the throat or lungs.

Protein 52

Protein Genetics Trials Allergies 52

pharmaphorum

SEPTEMBER 9, 2020

At the time, the Sanger Institute was working on the Deciphering Developmental Disorders study (DDD), which looked at the approximately 1-2% of the genome that is responsible for making proteins in patients with rare diseases. It’s essential to have hope in the future and in the promise that genomic medicine can bring.”.

Genome 80

Genome Genomics Genetics Genome Project 80

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

Roots Analysis

AUGUST 12, 2024

Molecular diagnostic solutions are pivotal across various medical fields, including oncological disorders, infectious diseases, genetic testing, and personalized medicine. Protein Based Assays- These tests measure the presence and levels of specific proteins, which can serve as biomarkers for various diseases.

Genetics 40

Genetics Gene Expression RNA DNA 40

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. The deal is worth $1.5

Life Science 98

Life Science RNA Vaccination Vaccine 98

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 Drugs Manufacturing 52

The Pharma Data

SEPTEMBER 22, 2020

About Zolgensma ® ( onasemnogene abeparvovec ) Zolgensma ® is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

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

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

The Pharma Data

AUGUST 3, 2021

SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Gene 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

XTalks

JANUARY 6, 2025

Whether its the integration of nanotechnology in medicine, the evolution of point-of-care (POC) diagnostics or the transformative impact of CRISPR and regenerative medicine, these biotech trends are pushing scientific boundaries and creating new opportunities for businesses and researchers alike. billion by 2032, with a CAGR of 10.1

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111