Roots Analysis

JANUARY 19, 2024

Introduction In a strategic move to accelerate the development and launch of genetic medicines, BridgeBio Pharma has successfully secured up to USD 1.25 In a deal worth USD 500 million in cash, BridgeBio Pharma has committed to providing the investors with a 5% royalty on worldwide net sales of its drug, acoramidis, pending FDA approval.

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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. It also affects Hispanic Americans, but at a lower prevalence. The mutation causes red blood cells to develop a crescent or “sickle” shape.

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XTalks

JANUARY 26, 2023

This is the fourth US FDA approval that BeiGene has received for Brukinsa since 2019. Diagnosis is confirmed by a differential complete blood count and genetic testing. Treatment selection is determined by age, disease severity, comorbidities and the presence or absence of certain genetic mutations.

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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.

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FDA Approval Gene Therapy Gene Clinical Trials 59

Drug Discovery World

JANUARY 23, 2023

“Put simply, radioresistant GBM cells prefer to stock up on fatty acids instead of utilising them as an energy source in order to reduce mitochondrial reactive oxygen species that may cause damage to their DNA, RNA, and proteins, and, in turn, cell death,” explains Prof Youn.

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XTalks

MARCH 10, 2023

This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher. It is caused by a mutation in the FXN gene , which is responsible for the production of the protein frataxin.

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XTalks

APRIL 11, 2024

After backing from the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) last month, Johnson and Johnson (J&J) and Bristol Myers Squibb (BMS) scored FDA approvals for the use of their B-cell maturation antigen (BCMA)-targeting CAR T-cell therapies as earlier line treatment options for multiple myeloma.

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STAT News

NOVEMBER 23, 2022

The approval of Hemgenix provides physicians with a new, potentially curative treatment option for patients with severe hemophilia B, a rare bleeding disorder caused by a genetic mutation that prevents the body from producing sufficient quantities of a clotting protein called Factor IX.

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The Pharma Data

APRIL 26, 2022

A drug used to treat asthma and allergies can bind to and block a crucial protein produced by the virus SARS-CoV-2, and reduce viral replication in human immune cells, according to a new study by researchers at the Indian Institute of Science (IISc). Targeting Nsp1 could therefore reduce the damage inflicted by the virus.

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Protein Drugs Allergies FDA Approval 52

pharmaphorum

MARCH 29, 2021

It is a CAR-T (chimeric antigen receptor T-cell) therapy, where a patient’s own T-cells are harvested, genetically modified and then reinjected to fight the cancer. However the payout worth up to $7 billion never happened after another CAR-T Breyanzi (lisocatbtagene maraleucel) was approved in February, missing a deadline set out in the CVR.

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The Pharma Data

AUGUST 13, 2020

NS Pharma has claimed a tentative FDA approval for its Viltepso (viltolarsen) injection in the treatment of Duchenne muscular dystrophy (DMD) in patients who are suitable to receive exon 53 skipping therapy. Mutations in the DMD gene can lead to an absence of the muscle cell protein dystrophin. of normal at baseline to 5.9%

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The Pharma Data

NOVEMBER 20, 2020

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies. Food and Drug Administration (FDA) has approved Zokinvy (lonafarnib) for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient Progeroid Laminopathies (PL). .

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pharmaphorum

NOVEMBER 12, 2021

Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients.

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The Pharma Data

MAY 31, 2022

About Evrysdi® (risdiplam) Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.

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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.

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Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. The mRNA constructs used in COVID-19 vaccines, for example, direct cells to produce a version of the “spike” protein studding the surface of SARS-CoV-2.

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XTalks

JUNE 28, 2023

The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial. DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness. It is injected into a patient’s muscle tissue.

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XTalks

NOVEMBER 30, 2023

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

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XTalks

MAY 1, 2023

Last week, the US Food and Drug Administration (FDA) granted accelerated approval to Biogen’s Qalsody (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 ( SOD1 ) gene. How Does Qalsody Work?

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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.

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XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.

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Roots Analysis

JANUARY 14, 2024

Given the ongoing scientific advancements and the rise of FDA-approved biologics, the pharmaceutical industry seems to be approaching the era of biologics. Insect cell lines have been frequently used as recombinant protein expression systems. They are easy to culture and are known to generate high yields of recombinant proteins.

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XTalks

FEBRUARY 24, 2023

The US Food and Drug Administration (FDA) granted accelerated approval to Genentech’s Lunsumio (mosunetuzumab) for adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) who have undergone two or more lines of systemic therapy. New treatment options are critically needed for FL.

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pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. Translarna has had European approval for usage since 2014 and has also been available under terms similar to the MAA to DMD patients in Scotland since 2021.

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XTalks

JUNE 15, 2022

The Novavax COVID-19 vaccine was recommended for emergency use in adults by the World Health Organization (WHO) in December 2021 , and is approved in over 40 countries. The Novavax candidate NVX-CoV2373 is a protein-based vaccine developed using recombinant nanoparticle technology. The Novavax COVID-19 Vaccine Candidate.

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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.

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pharmaphorum

SEPTEMBER 16, 2022

A mere six months ago Verily launched the study with Sosei Heptares – a global leader in GPCR structure-based drug design – with an aim to “prioritise protein targets for therapeutic targeting in immune-mediated disease”. What, then, is the solution? It also bodes well for improving clinical trial success rates. AI relies on data.

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XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. The approval came after a couple of setbacks, including a rejection in 2020 and a delay earlier this year.

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Drug Discovery World

MAY 24, 2023

G protein-coupled receptors (GPCRs) are a large family of proteins that regulate many processes in the body and are the target of one third of clinically used drugs. Several FDA-approved drugs that target the NK1 receptor are used to prevent nausea and vomiting associated with chemotherapy or surgery.

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Drug Discovery World

NOVEMBER 14, 2023

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP).

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Drug Discovery World

OCTOBER 18, 2023

As such, they offer highly stable expression of human genes, mRNA, and proteins. Food & Drug Administration (2019) FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. Accessed 26 Jul 23. Accessed 26 Jul 23.

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XTalks

AUGUST 17, 2020

As DMD is caused by a lack of functional dystrophin protein, which supports and strengthens muscle fibers, therapies designed to treat this rare genetic disorder have focused on exon skipping to avoid the area of the dystrophin gene that is mutated and produce a nearly full-length dystrophin protein.

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XTalks

SEPTEMBER 17, 2021

The ALS Association is pushing for rapid FDA approval of Amylyx Pharmaceuticals Inc.’s The complex is designed to target the toxic unfolded proteins in the endoplasmic reticulum, as well as mitochondrial dysfunction, which is being increasingly recognized as a pathogenic mechanism underlying the disease.

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XTalks

MARCH 6, 2024

This includes discussions on cutting-edge technologies and methodologies in brain research, such as neuroimaging, genetic engineering and artificial intelligence. Boston Scientific’s Deep Brain Stimulation (DBS) Software received FDA approval last year for the treatment of Parkinson’s disease and essential tremor.

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FDA Approval In-Vitro Research Marketing 69

XTalks

MARCH 28, 2024

For CRC, treatment options that are targeting genetic mutations such as BRAF , KRAS and HER2 have been developed. Gene Editing and CAR T-Cell Therapy Genetic engineering technologies, such as CRISPR-Cas9, are providing newer potential avenues for cancer treatment. Yang Liu and CFO Abid Ansari – Xtalks Life Science Podcast Ep.

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XTalks

JUNE 8, 2021

SMA is a rare genetic condition that leads to a loss of motor neurons that results in progressive muscle weakness and wasting, paralysis and, when left untreated in its most severe form, breathing difficulties leading to permanent ventilation or death for most patients by the age of two. With a price tag of over $2.5

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The Pharma Data

SEPTEMBER 28, 2021

In addition, results from Part 1 of Study SRP-9001-102, an ongoing, randomised, double-blind, placebo-controlled clinical test evaluating the security , efficacy, and tolerability of one dose of SRP-9001 in 41 boys with DMD, showed that the study met its primary biological endpoint of change in micro-dystrophin protein expression from baseline.

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