XTalks

DECEMBER 18, 2024

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.

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XTalks

NOVEMBER 10, 2023

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

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The Pharma Data

NOVEMBER 27, 2020

FDA Approves 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 genetic diseases of obesity, announced today that the U.S.

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FDA Approval Genetic Disease Genetics Clinical Trials 52

XTalks

MAY 26, 2023

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).

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Gene Therapy Gene Dermatology FDA Approval 97

XTalks

MAY 17, 2023

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.

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Pharmaceutical Technology

APRIL 24, 2023

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 FDA approved the first nanodrug, Doxil, for Kaposi sarcoma in 1995.

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Drug Delivery Vaccine Vaccination Research 147

XTalks

FEBRUARY 28, 2022

In the US, 25 to 30 million Americans, or one in ten, are living with a rare disease. Are Most Rare Diseases Genetic? While 72 percent of rare diseases are genetic, not all are. Rare genetic diseases can be caused by either somatic or germline genetic changes. The orphan drug designation was.

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Clinical Trials Clinical Trials Genetics Trials 98

XTalks

MAY 12, 2023

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.

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FDA Approval Genetics Trials Drugs 97

XTalks

AUGUST 6, 2020

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.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

XTalks

SEPTEMBER 25, 2024

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.

FDA Approval 52

FDA Approval Drugs Protein Gene 52

pharmaphorum

SEPTEMBER 28, 2022

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 genetic disease,” said Nia Tatsis, Vertex’ chief regulatory and quality officer, in a statement.

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Gene Editing Gene In-Vivo Clinical Trials 59

The Pharma Data

NOVEMBER 29, 2020

The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs. The FDA approved the drug on November 24. Typically, it is children who suffer from the disease. It was accepted for priority review on June 2.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

The Pharma Data

DECEMBER 8, 2020

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

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RNA Antibody Life Science Protein 52

XTalks

APRIL 1, 2025

Some individuals with hemophilia A may develop inhibitors against factor VIII, while those with hemophilia B can develop inhibitors against factor IX. Clinical Trials Backing Qfitlias Approval Two Phase III studies involving adult and pediatric male patients with hemophilia A or B supported Qfitlias FDA approval.

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FDA Approval RNA Clinical Trials Clinical Trials 59

XTalks

SEPTEMBER 17, 2020

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.

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Trials Gene Hormones Clinical Trials 98

XTalks

SEPTEMBER 25, 2024

NPC is a serious disease that leads to enormous adverse impacts on patients and families.

Genetics 52

Genetics Drugs Trials Pharmacy 52

XTalks

NOVEMBER 26, 2024

BridgeBio has been highly active in the genetic diseases 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.

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Genetic Disease Genetics Protein Clinical Development 105

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. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

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Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

SEPTEMBER 12, 2022

The FDA approval was based on data from the randomized, double-blind, placebo-controlled ASCEND study involving 31 adult patients. The FDA approval 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.

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FDA Approval Genetics Trials Gene 98

The Pharma Data

NOVEMBER 24, 2020

FDA Approves 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.

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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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Gene Therapy Gene Drugs FDA Approval 111

The Pharma Data

AUGUST 9, 2021

“Pompe disease is a rare genetic disease 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.

FDA Approval 52

FDA Approval Genetic Disease Genetics Drugs 52

XTalks

MARCH 19, 2025

Elevidys is the first FDA-approved gene therapy for DMD a rare genetic disorder characterized by progressive muscle degeneration. Young patients at higher doses developed severe liver toxicity that led to acute liver failure and a total of four deaths, prompting the FDA to place the trial on clinical hold.

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Gene Therapy Gene Genetics Clinical Trials 80

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.

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Gene Therapy Gene Trials Clinical Trials 95

XTalks

MARCH 3, 2021

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.

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FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

DECEMBER 29, 2022

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.

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Life Science Gene Therapy Gene Manufacturing 52

The Pharma Data

DECEMBER 20, 2020

Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S. The FDA approval 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.

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In-Vitro FDA Approval Drugs Manufacturing 52

XTalks

DECEMBER 22, 2021

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.

Life Science 98

Life Science RNA Vaccine Vaccination 98

pharmaphorum

JANUARY 10, 2022

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.

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Delveinsight

AUGUST 17, 2021

FDA’s Green Flag to Keytruda and Lenvima combination by Merck and Eisai for Advanced renal cell carcinoma (RCC). The FDA approved 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

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FDA Approval Medicine Vaccination Vaccine 98

XTalks

FEBRUARY 13, 2025

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.

FDA Approval 59

FDA Approval Genetics Clinical Trials Clinical Trials 59

XTalks

JANUARY 6, 2025

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 genetic diseases in the near future.

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The Pharma Data

NOVEMBER 25, 2020

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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