pharmaphorum

OCTOBER 5, 2022

The new drug candidate for FSHD will combine an RNA molecule from miRecule targeting double homeobox 4 (DUX4) – a protein that is mutated in FSHD – with a nanobody developed by Sanofi that targets muscle cells. billion deal in 2018.

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RNA Genetics Dermatology Antibody 105

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. Related: GW Pharmaceuticals’ CBD Drug Gains Second Approval.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

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.

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Vaccination Vaccine Gene Therapy RNA 98

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

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Protein RNA Production Genetics 52

pharmaphorum

AUGUST 1, 2022

The US biotech – which already has three antisense drugs for DMD on the market – had said earlier it was planning to delay the application into 2023, so the new announcement marks an acceleration of its plans. The three drugs required intravenous dosing once a week and work only in around a third of DMD patients.

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Gene Therapy Gene Genetics Trials 105

XTalks

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. In March, the US Food and Drug Administration (FDA) approved Daybue (trofinetide) , the first and only treatment for Rett syndrome. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.

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FDA Approval Genetics Trials Drugs 97

XTalks

SEPTEMBER 17, 2020

Surrogate endpoints were used as the basis for approval of 45 percent of new drugs reviewed by the FDA between 2010 and 2012. For example, studies of heart attack prevention medications use the surrogate endpoint of low-density lipoprotein cholesterol (LDL-C) to assess the effectiveness of new drugs. Cushing’s Disease.

Trials 98

Trials Gene Hormones Clinical Trials 98

pharmaphorum

JANUARY 10, 2022

The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for liver-targeted diseases.

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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). The drug’s flexible administration allows for use with or without food.

FDA Approval 52

FDA Approval Drugs Protein Gene 52

XTalks

SEPTEMBER 16, 2024

The US Food and Drug Administration (FDA) has accepted KalVista Pharmaceuticals’ New Drug Application (NDA) for sebetralstat, a novel, investigational oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE) attacks in adult and pediatric patients aged 12 years and older.

Protein 52

Protein Genetics Trials Allergies 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. Remix Therapeutics .

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RNA Antibody Life Science Protein 52

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. Efforts to improve SMA detection and treatment are gaining momentum.

Trials 52

Trials Antibody Genetics 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

Roots Analysis

AUGUST 12, 2024

Protein Based Assays- These tests measure the presence and levels of specific proteins, which can serve as biomarkers for various diseases. These tests can aid in the diagnosis and treatment monitoring of various conditions by providing information about the underlying biological processes and disease states.

Genetics 40

Genetics Gene Expression RNA DNA 40

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. Be Biopharma .

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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. The Food and Drug Administration (FDA) granted orphan-disease designation and breakthrough therapy designation in the U.S.,

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

XTalks

FEBRUARY 27, 2023

Chiesi Global Rare Diseases recently announced in a press release that Lamzede (velmanase alfa-tycv) received US Food and Drug Administration (FDA) approval for the treatment of non-neurological manifestations of alpha-mannosidosis in both adult and pediatric patients.

FDA Approval 52

FDA Approval Life Science Containment Genetics 52

The Pharma Data

OCTOBER 28, 2020

These SAEs were assessed as mild or moderate in severity and have generally improved over a period of a few weeks while disease domain improvements have been sustained for three months. The administration method will be modified to help reduce local drug contact time. Angelman syndrome is often misdiagnosed as autism or cerebral palsy.

Protein 40

Protein Production Development Drugs 40

XTalks

NOVEMBER 26, 2024

The US Food and Drug Administration (FDA) has approved Attruby (acoramidis) , marking a significant advancement for patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). This progressive disease occurs when misfolded transthyretin (TTR) proteins form amyloid deposits in the heart, leading to cardiac dysfunction.

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Genetic Disease Genetics Protein Clinical Development 105

The Pharma Data

JANUARY 9, 2021

The most common adverse events associated with valoctocogene roxaparvovec occurred early and included transient infusion-associated reactions and transient, asymptomatic, and mild to moderate rise in the levels of certain proteins and enzymes measured in liver function tests with no long-lasting clinical sequelae. GENEr8-1 Study Description.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing. Gene Therapy Definition.

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XTalks

APRIL 1, 2025

XTALKS WEBINAR: Keys to Success in Clinical Trials: A Strategic Guide for Biotechs and Startups Live and On-Demand: Thursday, May 22, 2025 , at 11am EDT (5pm CEST / EU-Central) Register for this free webinar to learn how biotechs can navigate endpoint challenges in clinical trials and accelerate their path to drug approval.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

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

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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Gene Therapy Gene FDA Approval In-Vivo 98

The Pharma Data

DECEMBER 3, 2020

Antiepileptic Drugs / 7B. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological Na V 1.1

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

The Pharma Data

MARCH 16, 2021

Food and Drug Administration (FDA) has accepted and granted priority review for a New Drug Application (NDA) for the hypoxia-inducible factor-2 alpha (HIF-2?) in patients with VHL disease-associated RCC. The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of September 15, 2021.

Drugs 52

Drugs Trials Genetics Protein 52

The Pharma Data

NOVEMBER 8, 2020

Food and Drug Administration (FDA) in terms of PDUFA dates. The drug is an investigational, novel, once-daily, oral atypical antipsychotic drug candidate designed to improve the efficacy of olanzapine while decreasing olanzapine-associated weight gain. November is promising to be a busy month for the U.S.

Trials 52

Trials Protein Gene Genetic Disease 52

The Pharma Data

DECEMBER 20, 2020

Food and Drug Administration (FDA) is plenty busy with COVID-19 vaccine Emergency Use Authorizations (EUAs) this month, but they’re also wrapping up the year with a few PDUFA dates for other therapies. The drug is a once-daily, beta-3 adrenergic agonist. Here’s a look. Urovant Sciences’ Vibegron for Overactive Bladder.

In-Vitro 52

In-Vitro FDA Approval Drugs Manufacturing 52

The Pharma Data

NOVEMBER 24, 2020

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. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction.

FDA Approval 52

FDA Approval Production RNA Medicine 52

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. Novartis today announced that the U.S.

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Clinical Trials Clinical Trials Gene Therapy Gene 52

XTalks

DECEMBER 13, 2023

Fulgent Genetics continues to make strides in its core genetics business, maintaining momentum in three primary areas: precision diagnostics, anatomic pathology and pharmaceutical services. This substantial investment is accelerating the growth of their new drug pipeline. million and $38.8 million and $33.9 million and $38.8

Genetics 111

Genetics Vaccination Vaccine DNA 111

The Pharma Data

SEPTEMBER 22, 2020

Basel, September 23 , 2020 – Novartis Gene Therapies recently received feedback from the US Food and Drug Administration (FDA) following their review of data from the STRONG study of the intrathecal (IT) formulation of AVXS-101 in older patients with spinal muscular atrophy (SMA). More than 30% of patients with SMA Type 2 will die by age 25.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 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

pharmaphorum

JANUARY 10, 2022

The first mRNA-based vaccines were brought onto the market in 2020, with Pfizer/BioNtech and Moderna receiving emergency use authorisation from the US Food and Drug Administration (FDA) for the prevention of COVID-19. The breakthrough was hailed as a revolution for disease treatment and particularly a major success against the pandemic.

RNA 111

RNA Vaccination Vaccine Pharma Companies 111

Delveinsight

JANUARY 12, 2021

Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor cells. Thus, the drug appears to be a promising therapeutic agent in treating different forms of cancer in combination with other drugs.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

The Pharma Data

DECEMBER 27, 2020

The drug, Yeliva (opaganib) is a first-in-class, oral spingosine kinase-2 (SK2) selective inhibitor with triple-action effect on the pathophysiological processes linked to COVID-19. The drug is a humanized IgG4 monoclonal antibody that blocks CCR5 and is being developed for COVID-19, HIV and metastatic triple-negative breast cancer.

Trials 52

Trials Antibody Gene Therapy Gene 52

XTalks

FEBRUARY 13, 2025

HAE is a rare genetic disorder characterized by recurrent episodes of severe swelling (angioedema) in various parts of the body, including the skin, gastrointestinal tract and airways. The FDA has set a Prescription Drug User Fee Act (PDUFA) target date of June 17, 2025, for its review decision.

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Trials Genetics Allergies Drugs 82