pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels.

Genetic Disease 104

Genetic Disease Protein RNA Doctor 104

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

XTalks

DECEMBER 13, 2023

Modern is making significant strides in advancing its mRNA technology and diversifying its product portfolio to address a broad spectrum of medical conditions. The company is unwavering in its commitment to creating and introducing innovative vaccines that safeguard the global population against infectious diseases. million and $18.0

Genetics 111

Genetics Vaccination Vaccine DNA 111

The Pharma Data

OCTOBER 14, 2020

Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases.

Protein 52

Protein RNA Production Genetics 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). As a topical treatment, it is also the first readily redosable gene therapy.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. The disease is caused by mutations on the TSC1 and TSC2 genes, which produce the proteins hamartin and tuberin, respectively.

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.

Vaccine 98

Vaccine Vaccination Gene Therapy RNA 98

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.

RNA 105

RNA Genetics Dermatology Antibody 105

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. Besides, BeiGene has an option to co-detail the product in North America, which will be funded in part by Novartis. and Europe.

Licensing 52

Licensing Licensing Antibody Gene Therapy 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. This not only allowed Pfizer to establish a COVID-19 vaccine but the company also recently announced that it had begun a study testing an mRNA flu vaccine.

RNA 111

RNA Vaccination Vaccine Pharma Companies 111

pharmaphorum

AUGUST 1, 2022

Sarepta’s gene therapy – like rivals from Pfizer and Solid Biosciences – codes for a shortened form of the dystrophin protein that is deficient in patients with the X-linked muscle-wasting disease, which occurs primarily in males.

Gene Therapy 105

Gene Therapy Gene Genetics Trials 105

XTalks

DECEMBER 13, 2023

The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells. Obenshain said the company will book revenue when the final product is infused, and that the product manufacturing could take 70 to 105 days.

Gene Therapy 98

Gene Therapy Gene FDA Approval In-Vivo 98

The Pharma Data

DECEMBER 3, 2020

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

XTalks

SEPTEMBER 17, 2020

In the case of the former, pharmaceutical and biologics companies would be eligible for approval of their product through traditional regulatory pathways; the latter could support accelerated approval. Symptoms and Etiology: Cushing’s disease is a disorder caused by excess cortisol which is often the result of a pituitary adenoma.

Trials 98

Trials Gene Hormones Clinical Trials 98

The Pharma Data

DECEMBER 8, 2020

Now poised to advance a robust therapeutics pipeline to clinical development, Nuance will use the funds for ongoing R&D of existing products and business development of potential new assets. Through its Shielded Living Therapeutics platform, the company is developing functional cures for chronic diseases. Sigilon Therapeutics .

RNA 52

RNA Antibody Life Science Protein 52

XTalks

JANUARY 3, 2025

Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism. By lowering its production, Tryngolza significantly reduces triglyceride levels in the blood. 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

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

According to the World Health Organization, there are over 40,000 in vitro diagnostics products available in the market. This vast array of products highlights the critical role of diagnostics in healthcare. It is worth mentioning that molecular diagnostics is a subset of the broader in vitro diagnostics market.

Genetics 40

Genetics Gene Expression RNA DNA 40

The Pharma Data

NOVEMBER 8, 2020

Based on the feedback from our Type A meeting in February, we conducted additional product testing with the goal of addressing the Chemistry, Manufacturing and Controls deficiencies discussed in the CRL.”. “We have been encouraged by our interactions with the FDA following our CRL and are pleased to resubmit our Zimhi NDA,” said Dennis J.

Trials 52

Trials Protein Gene Genetic Disease 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

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

JULY 6, 2021

Roche’s Chief Medical Officer and Head of Global Product Development. 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. Hemlibra is a bispecific factor IXa- and factor X-directed antibody.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

The Pharma Data

DECEMBER 20, 2020

Klisyri is a home-grown product discovered and characterized by Athenex scientists and developed from pre-IND to NDA by the Athenex team. The FDA also had questions regarding testing, labeling, and features of the combination product that were not related to the drug constituent.

In-Vitro 52

In-Vitro FDA Approval Drugs Manufacturing 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. The deal is worth $1.5

Life Science 98

Life Science RNA Vaccination Vaccine 98

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. Nor can there be any guarantee that such products will be commercially successful in the future.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Gene 52

The Pharma Data

DECEMBER 27, 2020

The drug is a topical ointment applied to the lower lid to address the build-up and shedding of proteins at the opening of the Meibomian gland. GA Depot is a long-acting injection of the approved Glatiramer Acetate products (Copaxone and its generic formulations). The drug is an autologous IL13Ralpha2-CAR-T cell product.

Trials 52

Trials Antibody Gene Therapy Gene 52

The Pharma Data

OCTOBER 28, 2020

The interim analysis did not include data from other exploratory outcome measures such as seizure frequency, sleep diaries, EEG patterns, UBE3A protein levels in the CSF, ambulation by wearable device, and adaptive behaviors. This condition is typically not inherited but instead occurs spontaneously. About Ultragenyx.

Protein 40

Protein Production Development Drugs 40

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

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

XTalks

JANUARY 6, 2025

Medical device manufacturers are leveraging these technologies to enhance their products, supporting healthcare providers and improving patient outcomes. Moreover, AI has the potential to identify features and signs of disease that may be overlooked by human experts, offering a new layer of diagnostic capability.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111

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. Without proper treatment, HAE attacks can be painful, debilitating and potentially life-threatening, especially if they involve the airway.

Trials 82

Trials Genetics Allergies Drugs 82