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Mirum Pharmaceuticals, a biotech innovator known for developing therapies for rare metabolic disorders, now has FDAapproval for its new treatment, Ctexli (chenodiol) tablets the first and only medication approved for cerebrotendinous xanthomatosis (CTX) in adults.
The US Food and Drug Administration (FDA) has approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator, to treat cystic fibrosis (CF) in patients aged six years and older with at least one F508del mutation or another responsive CFTR mutation.
BridgeBio has been highly active in the geneticdiseases space. Its investigational therapy infigratinib recently received Breakthrough Therapy designation, becoming the first-ever potential treatment for achondroplasia — a genetic condition affecting bone growth — to achieve this milestone.
It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious geneticdisease affecting the skin and mucosal tissues.
The FDAapproval was based on data from the randomized, double-blind, placebo-controlled ASCEND study involving 31 adult patients. The FDAapproval came ahead of its October 3 target date, which had been extended by three months. Patients in the trial received Xenpozyme or placebo for 52 weeks.
Venture capitalists have poured $42 billion into drug development over the past three years. Most small biotech companies rely on venture capitalist funding to develop new drugs but is that a good way to go? billion, which was much lower than previous studies, which have placed the average cost of drug development as $2.8
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. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).
In 2013, the US Food and Drug Administration (FDA) approved Ravicti (glycerol phenylbutyrate), manufactured by Hyperion Therapeutics, which has since then been acquired by Horizon Pharma. In order to find the specific RNA sequences for a geneticdisease, the biotech is using an AI- and machine learning-based approach.
While manageable, the condition requires lifelong treatment and monitoring to prevent complications and ensure healthy development. CAH affects approximately one in 15,000 live births worldwide, and an estimated 30,000 people are living with the condition in the US.
“Pompe disease is a rare geneticdisease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, M.D., deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research.
Acute manifestations include thrombocytopenia, microangiopathic hemolytic anemia, bleeding episodes, headache and abdominal pain, while chronic ones include stroke, cardiovascular disease and damage to organs. As a rare disease, cTTP affects less than 1,000 individuals in the US. “As
FDAApproves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency. Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare geneticdiseases of obesity, announced today that the U.S.
Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB).
FDAApproves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
The Bespoke Gene Therapy Consortium was jointly launched by the NIH and US Food and Drug Administration (FDA) in 2021 to help address common issues that gene therapy developers face. It included formulating a standardized therapeutic development model that would help accelerate clinical trial processes for new treatments.
With the two approvals acting as a validation of the technology, larger companies are prepared to make larger investments to ensure that they are at the forefront of developments in RNA-focused therapies. Both biotechs focus on mRNA therapies but not just for infectious diseases.
In the US, 25 to 30 million Americans, or one in ten, are living with a rare disease. Are Most Rare DiseasesGenetic? While 72 percent of rare diseases are genetic, not all are. Rare geneticdiseases can be caused by either somatic or germline genetic changes. The orphan drug designation was.
The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Rett syndrome is characterized by a distinctive pattern of symptoms that gradually emerge after a period of seemingly normal development. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.
The US Food and Drug Administration (FDA) has approved the use of an oral cannabidiol (CBD) solution called Epidiolex for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients aged one year and older. Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant.
In a significant regulatory milestone, the US Food and Drug Administration (FDA) approved Aqneursa (levacetylleucine) for treating neurological symptoms associated with Niemann-Pick disease type C (NPC). NPC, caused by mutations in the NPC1 or NPC2 gene, typically shortens life expectancy to around 13 years.
The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market. “We continue to work with urgency to bring forward the first CRISPR therapy for a geneticdisease,” said Nia Tatsis, Vertex’ chief regulatory and quality officer, in a statement.
The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. The approval was granted to BridgeBio Pharma, Inc.
The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. They can be developed and adapted quickly. This did the trick and set the stage for the development of viable mRNA-based therapeutics. RNA Therapeutics.
The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs. The FDAapproved the drug on November 24. Typically, it is children who suffer from the disease. It was accepted for priority review on June 2.
Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S. The FDAapproval of Klisyri is a significant milestone for Athenex,” said Johnson Lau, chairman and chief executive officer of Athenex. and Europe, including Russia. Most Read Today.
AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Sigilon Therapeutics
While the table lists acceptable surrogate endpoints for trials of more common conditions, such as asthma, type 2 diabetes and various cancer types, the document also provides key information to developers of certain rare disease therapies. Novartis’ Signifor (pasireotide) is also indicated in the treatment of the disease.
FDA’s Green Flag to Keytruda and Lenvima combination by Merck and Eisai for Advanced renal cell carcinoma (RCC). The FDAapproved the combination of Keytruda and Lenvima produced by Merck and Eisai, as a first-line treatment of adult patients with advanced renal cell carcinoma (RCC). The rarity of disease – 1.36
For the first time in the US, adults living with familial chylomicronemia syndrome (FCS) have an FDA-approved treatment option. Investigational drug plozasiran , developed by Arrowhead Pharmaceuticals, is under review for FCS treatment. In clinical trials, it showed up to an 86 percent reduction in triglyceride levels.
As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies. In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light.
Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare geneticdiseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.
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).
In 1974, Dr. Robert Langer was part of the first cohort of researchers to begin nanoparticle research , as his lab at Cornell University developed tiny particles to deliver large molecules for angiogenesis. However, after a period of slow movement in the field, the FDAapproved the first nanodrug, Doxil, for Kaposi sarcoma in 1995.
This blog highlights the top 10 biotech trends for 2025, showcasing cutting-edge technologies and approaches that promise to redefine patient care, accelerate drug development and expand our understanding of human biology. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming geneticdiseases in the near future.
The FDA has granted approval to SpringWorks Therapeutics Gomekli (mirdametinib) for the treatment of adult and pediatric patients aged two years and older with neurofibromatosis type 1 (NF1). NF1 is a rare genetic disorder characterized by the development of tumors along nerves in the skin, brain and other parts of the body.
The mRNA vaccine candidate developed by Pfizer and Germany-based BioNTech demonstrated 95% efficacy in clinical trials. Food and Drug Administration (FDA) will review the data on Dec. The vaccine approvals are the proverbial light at the end of the tunnel that is COVID-19.
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