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. One patient developed fatal multi-organ damage and one patient with fatal bronchopulmonary aspergillosis, which were related to the extreme immune reaction.

FDA Approval 85

FDA Approval Genetics Protein Antibody 85

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.

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 52

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.

FDA Approval 52

FDA Approval Manufacturing Trials Clinical Trials 52

XTalks

MARCH 10, 2023

But with Skyclarys (omaveloxolone), the first and only drug to treat Friedreich’s ataxia in patients aged 16 years and older, Reata Pharmaceuticals has developed a new treatment option to improve neurological function. This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher.

FDA Approval 52

FDA Approval Engineer Clinical Trials Clinical Trials 52

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”. Kim recognised then, he said, that better tools for medicine needed to be developed, and that they still do.

Development 135

Development Research Drugs Clinical Trials 135

STAT News

NOVEMBER 23, 2022

Food and Drug Administration on Tuesday approved the first gene therapy to treat people with hemophilia B, an inherited bleeding disorder , STAT writes. The one-time treatment, called Hemgenix, was developed by the Dutch biotech company UniQure and will be marketed by CSL Behring, an Australian pharmaceutical company.

Gene Therapy 97

Gene Therapy FDA Approval Gene Genetics 97

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.

Protein 52

Protein Drugs Allergies FDA Approval 52

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%

FDA Approval 52

FDA Approval Genetics Protein Regulation 52

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. The tool meanwhile could also be applied to new outbreaks of other novel viral infections as they emerge, according to the scientists.

Doctors 135

Doctors Doctor Drugs Gene Expression 135

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

The Pharma Data

NOVEMBER 20, 2020

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies. Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that the U.S. PALO ALTO, Calif., ABOUT ZOKINVY (LONAFARNIB).

FDA Approval 52

FDA Approval Genetics Clinical Trials Clinical Trials 52

The Pharma Data

MAY 31, 2022

. “The priority review and subsequent approval of Evrysdi for babies under two months of age speaks to the urgent ongoing need for additional treatment options for babies with SMA,” said Levi Garraway, M.D., Roche’s Chief Medical Officer and Head of Global Product Development. Food and Drug Administration in 2017.

FDA Approval 52

FDA Approval Protein In-Vitro Medicine 52

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. The recommended dosage of Qalsody is 100 mg (15 mL) per administration.

FDA Approval 89

FDA Approval Gene Genetics Protein 89

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.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

XTalks

NOVEMBER 30, 2023

Global entities such as the WHO, the Global Fund and the Joint United Nations Programme on HIV/AIDS (UNAIDS) have developed comprehensive strategies aligned with Sustainable Development Goal (SDG) target 3.3, This marked the first FDA-approved injectable, complete regimen for HIV-infected adults, administered once a month.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

The Pharma Data

NOVEMBER 30, 2020

(Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today provided an update on its COVID-19 vaccine program. About NVX-CoV2373 NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of SARS-CoV-2, the virus that causes COVID-19 disease.

Clinical Development 52

Clinical Development Vaccination Vaccine Development 52

Roots Analysis

JANUARY 14, 2024

With the increased interest and gradual shift of investment from small molecule drugs to biologics and the establishment of several biologics manufacturing companies / biologics CMOs, more than 250 biologic therapies and vaccines have been developed, globally. Mammalian cell cultures offer stability and are preferred for long term usage.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

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.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

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.

Gene Therapy 98

Gene Therapy Gene FDA Approval DNA 98

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. Why is the approval of Lunsumio a significant milestone for Genentech and Roche?

FDA Approval 52

FDA Approval Drugs Antibody Medicine 52

Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. As such, they offer highly stable expression of human genes, mRNA, and proteins.

Gene Therapy 64

Gene Therapy Gene Clinical Trials Clinical Trials 64

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.

Vaccination 59

Vaccination Vaccine Immune Response Protein 59

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

Drug Discovery World

JUNE 1, 2023

According to NCBI, most of the ADCs developed so far are for use in the treatment of cancer, but there is plenty of potential for using ADCs to treat other diseases 1. A Nature publication confirmed that there are currently 12 FDA-approved ADCs on the market, and nine of these secured FDA approval in the past six years 2.

Antibody 98

Antibody Marketing Drugs Licensing 98

XTalks

MARCH 6, 2024

This includes discussions on cutting-edge technologies and methodologies in brain research, such as neuroimaging, genetic engineering and artificial intelligence. On the other, the development of smaller, portable MRI machines is favored for their ease and convenience.

FDA Approval 69

FDA Approval In-Vitro Research Marketing 69

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). Without treatment, cTTP is ultimately fatal.

FDA Approval 52

FDA Approval Gene Clinical Trials Clinical Trials 52

XTalks

MARCH 28, 2024

Colorectal Cancer: Advances in Treatment Options In recent years, there has been considerable progress in understanding the biology of CRC, which has helped develop more effective and personalized treatment options. An overview of some of the most promising developments in CRC treatment is provided below.

Gene Editing 59

Gene Editing Life Science Clinical Trials Clinical Trials 59

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.

Research 52

Research Drugs Protein FDA Approval 52

XTalks

AUGUST 17, 2020

Duchenne muscular dystrophy (DMD) patients with a mutation in exon 53 of the dystrophin gene now have a second approved treatment option, and Xtalks spoke with Sharon Hesterlee, PhD, Muscular Dystrophy Association executive vice president and chief research officer, to learn more.

Drugs 105

Drugs Clinical Trials Clinical Trials Gene 105

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.

Gene Therapy 98

Gene Therapy Gene FDA Approval Antibody 98

XTalks

FEBRUARY 5, 2024

This article serves as a resource for healthcare professionals, life sciences researchers and industry enthusiasts seeking to stay informed of the latest developments and success stories in oncology therapeutics. Pharmacyclics requested to voluntarily withdraw the approvals and decided to forgo an opportunity for a hearing.

FDA Approval 101

FDA Approval Drugs Sales Development 101

The Pharma Data

SEPTEMBER 28, 2021

Roche’s Chief medic and Head of worldwide development. Our goal is to still lead the way in developing transformative medicines for neuromuscular diseases. We are grateful for the partnerships that are helping us to develop new therapies for people impacted by these devastating rare diseases.”. About SRP-9001 SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)

Gene Therapy 52

Gene Therapy Protein Gene Clinical Trials 52

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

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

XTalks

DECEMBER 4, 2023

The FDA said it is investigating the identified risk of developing the T-cell malignancies as secondary cancers with serious outcomes, including hospitalization and death, and also looking at the need for regulatory action. Novartis and BMS echoed similar sentiments regarding their respective therapies.

Gene Therapy 69

Gene Therapy Gene Clinical Trials Clinical Trials 69

The Pharma Data

NOVEMBER 9, 2020

(Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for NVX-CoV2373, the Company’s COVID-19 vaccine candidate. President of Research and Development, Novavax.

Vaccination 52

Vaccination Vaccine Clinical Trials Clinical Trials 52

XTalks

FEBRUARY 5, 2024

This article serves as a resource for healthcare professionals, life sciences researchers and industry enthusiasts seeking to stay informed of the latest developments and success stories in oncology therapeutics. Pharmacyclics requested to voluntarily withdraw the approvals and decided to forgo an opportunity for a hearing.

FDA Approval 52

FDA Approval Drugs Sales Development 52

Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

Genetics 52

Genetics Research Medicine Clinical Research 52