Broad Institute

OCTOBER 11, 2023

He also read everything he could about her disease, including emerging evidence that suggested that the immune system could recognize and kill Merkel cell carcinoma. His mother had a presentation of the disease that suggested her immune system was already on the job. This is what we need for patients,” Manguso said.

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The Pharma Data

AUGUST 20, 2020

Invasive Meningococcal Disease (IMD) is uncommon, with country-specific reported cases ranging from 0.1 cases per 100,000 population in 2017. The investigational vaccine has the potential to reduce the number of injections and thereby improve completion rates.”.

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The Pharma Data

JUNE 18, 2021

First approval of a conjugate vaccine that helps protect against 20 serotypes responsible for the majority of invasive pneumococcal disease and pneumonia, 1,2,3,4,5,6,7 including seven responsible for 40% of pneumococcal disease cases and deaths in the U.S. Following today’s FDA approval, the U.S. Jansen, Ph.D.,

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The Pharma Data

MAY 18, 2021

Food and Drug Administration (FDA) approval of the VENTANA MMR RxDx Panel for advanced or recurrent endometrial cancer patients. FDA approval of the VENTANA MMR RxDx Panel provides clinicians with access to a fully automated, easy-to-use MMR test to identify patients who are eligible for therapy with JEMPERLI.

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The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

Disease 52

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The Pharma Data

MAY 31, 2022

Without treatment, infants do not achieve these milestones in the natural history of the disease. “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.,

FDA Approval 52

FDA Approval FDA Treatment Clinical Trials 52

Agency IQ

MAY 17, 2024

The Creating Hope Reauthorization Act would reauthorize the FDA’s Rare Pediatric Disease Priority Review Voucher program. The markup hearing comes three months after the E&C Committee last held a hearing on 18 bills related to rare diseases, including all four of the above-mentioned bills. DORIS MATSUI (D-Calif.)

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Drug Target Review

SEPTEMBER 19, 2023

The chips can be modelled with primary cells, IPS derived stem cells or patient derived cells and potentiates to unlock molecular mechanisms that drive the disease pathophysiology, holding a greater promise. The first successful chip adaptation to a lung model was first described in 2010 by Donald Ingber, a bioengineer at Wyss institute.

Animal Testing 98

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New Drug Approvals

OCTOBER 24, 2023

Molecular Weight: 631.700 FDA APPROVED, To treat moderately to severely active ulcerative colitis in adults, 10/12/2023 Velsipity Etrasimod , sold under the brand name Velsipity , is a medication that is used for the treatment of ulcerative colitis (UC). [1] Peyrin-Biroulet L, Christopher R, Behan D, Lassen C (May 2017).

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The Pharma Data

NOVEMBER 15, 2020

” The study looked at more than 301,000 antipsychotic prescriptions filled between 2007 and 2017 for children ages 2 to 7 who were covered by private insurance. ” Antipsychotic medication comes with risks to children, including weight gain, sedation, diabetes, high cholesterol, cardiovascular disease and unexpected death. .

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Alta Sciences

APRIL 17, 2024

Gene Therapy Gene therapy is currently considered for diseases, often rare, that have no other cures. We have conducted over 130 in-life studies in the last five years (over 95% conducted in NHPs), including IND-enabling studies for rare diseases and CRISPR gene editing therapeutics in NHPs.

Drug Development 52

Drug Development Treatment FDA Approval Therapies 52

The Pharma Data

AUGUST 22, 2021

In CheckMate -274, Opdivo nearly doubled median disease-free survival compared to placebo in the intent-to-treat population 1. Opdivo is now approved in earlier stages of disease for three types of cancer, including the first and only PD-1 inhibitor approved for urothelial carcinoma in the adjuvant setting. to 13.9]).

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Drug Target Review

AUGUST 29, 2024

MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs. What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies?

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Vial

DECEMBER 5, 2023

Among others, NGS has led to the identification of disease-causing variants and novel drug targets and an improved understanding of complex biological events, e.g., the heterogeneity of tumors. 2022) recommend matching interventions to specific patient characteristics should be implemented early in the disease course. Fountzilas et al.

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Drug Target Review

JULY 5, 2024

Since brain organoids can be derived from either diseased or healthy patients, they can recapitulate human (patho)physiology making them a better choice compared to mammalian cell systems that often fail to recapitulate the human phenotype. 545:54-59 (2017). Cerebral organoids as tools to identify the developmental roots of autism.

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Targeted Protein Degradation Drugs Disease FDA Approval 64

The Pharma Data

APRIL 4, 2022

The target action date for the FDA decision on this investigational use is August 3, 2022. FDA granted Breakthrough Therapy designation to Dupixent for the treatment of patients aged 12 years and older with EoE. Dupixent was also granted Orphan Drug designation for the potential treatment of EoE in 2017. In the U.S.,

FDA 52

FDA Treatment Disease FDA Approval 52

The Pharma Data

DECEMBER 8, 2020

Some of the funds will be used for the development of therapeutics for oncology and other serious diseases through its proprietary ImmunoTAC technology platform. The ImmunoTAC platform pairs proprietary payloads that modulate key disease modifying pathways with monoclonal antibodies directed at specific disease sites.

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NIH Director's Blog: Drug Development

JULY 20, 2017

Credit: Brittany Mahoney As NIH Director, I often hear stories of how people with serious diseases—from arthritis to Zika infection—are benefitting from the transformational power of NIH’s investments in basic science. Cystic Fibrosis Foundation News Release, July 18, 2017. First, a bit of history! About 30,000 Americans have CF.

Clinical Trials 52

Clinical Trials Small Molecule Therapies Disease 52

Vial

JUNE 12, 2024

In clinical trials, PROs may assess disease progression, consequence(s) of the intervention being studied, functional status, and social, mental, and emotional limitations. PROs can be disease-specific, condition-specific, or generic. Generic PROs are used for any disease or population. For HF, Savarese et al.

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Clinical Trials Trials FDA Clinical Research 52

The Pharma Data

SEPTEMBER 28, 2021

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 SMA SMA may be a severe, progressive neuromuscular disease which will be fatal.

Clinical Trials 52

Clinical Trials Protein Expression Therapies FDA 52

The Pharma Data

OCTOBER 15, 2020

John Hospital, researchers compared 649 patients from two cohorts: a recent cohort (2017–2019) from the RECOVER III post-market approval (PMA) study, after the widespread adoption of the best practice of placing Impella pre-PCI, and a cohort from before PMA (2008–2014) when the practice of placing Impella pre-PCI was not yet widely adopted.

Hospitals 52

Hospitals FDA Approval FDA Treatment 52

The Pharma Data

JANUARY 31, 2021

Many major disease targets are trimerization-dependent such as the tumor necrosis factor (TNF) superfamily (involved in extrinsic apoptosis, immune co-stimulation and inflammation) as well as enveloped RNA virus antigens responsible for entry into host cells. . CHENGDU, China , Feb. About Trimer-Tag© Technology. About CEPI.

Clinical Trials 52

Clinical Trials Vaccine Trials Virus 52

Drug Target Review

AUGUST 3, 2023

Clinical trials are now underway for various genetic subtypes of both rare and common diseases, on the understanding that medications utilising genetic biomarkers have a significantly higher chance of success. There are clinical trials underway for genetic subtypes of rare and common diseases. To date, the company has raised $14.9

DNA 52

DNA Clinical Trials Trials Disease 52

Drug Target Review

DECEMBER 5, 2024

1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2. 2 Since then, the FDA has approved four more AAV-based gene therapies—Zolgensma, Hemgenix, Elevidys and Rocktavian—for treating various diseases.

Therapies 52

Therapies Virus DNA Compliance 52

The Pharma Data

DECEMBER 3, 2020

Many major disease targets are trimerization-dependent such as the tumor necrosis factor (TNF) superfamily (involved in extrinsic apoptosis, immune co-stimulation and inflammation) as well as enveloped RNA virus antigens responsible for entry into host cells. About Trimer-Tag © Technology. For more information, visit www.dynavax.com.

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Vaccine Immune Response Virus RNA 52

The Pharma Data

JANUARY 21, 2021

I can only hope that their STS experience further inspires them to take on and help solve the biggest challenges facing mankind – from climate change to disease and future pandemics.” About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases.

Science 40

Science Engineering Molecular Biology Bioinformatics 40

New Drug Approvals

AUGUST 26, 2023

Zuranolone CAS 1632051-40-1 Zurzuvae FDA APPROVED 8/4/2023, To treat postpartum depression Press Release WeightAverage: 409.574 Monoisotopic: 409.272927379Chemical FormulaC 25 H 35 N 3 O 2 SAGE 217 SAGE-217 SAGE217 Zuranolone , sold under the brand name Zurzuvae , is a medication used for the treatment of postpartum depression. [1]

FDA 52

FDA FDA Approval Clinical Trials Treatment 52

The Pharma Data

JUNE 18, 2021

BOTOX ® Cosmetic is the first and only product of its kind FDA-approved to treat three areas, temporarily improving the appearance of moderate to severe forehead lines, glabellar lines and crow’s feet in adults. Department of Health and Human Services FDA Approval Letter for BOTOX® Cosmetic for Glabellar Lines, 2002.

Doctors 52

Doctors FDA Approval Treatment Production 52

FDA Law Blog: Biosimilars

FEBRUARY 29, 2024

The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval. If the confirmatory trial shows that the drug actually provides a clinical benefit, then the FDA grants traditional approval for the drug. 8,128,929 [] for [PTE] request under 35 U.S.C. §

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FDA Production FDA Approval Licensing 59

The Pharma Data

SEPTEMBER 10, 2021

More than 20 abstracts across disease states, including Parkinson’s disease, spasticity and cervical dystonia, will be presented. “Our research presented at MDS 2021 builds upon our expertise in neuroscience and reinforces our mission to advance the standards of care for people living with these debilitating diseases.”

Doctors 52

Doctors Clinical Trials Disease Therapies 52

The Pharma Data

JUNE 18, 2021

“The results add to the extensive clinical evidence supporting the use of single-agent ibrutinib for long-term disease control.” 2.2017 were used given 74 percent enrollment in 2017. IMBRUVICA ® is now approved in 101 countries and has been used to treat more than 230,000 patients worldwide across its approved indications.

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FDA Approval Treatment Therapies FDA 40

The Pharma Data

JULY 23, 2021

We took this action following the Agency’s industry-wide evaluation of accelerated approvals for checkpoint inhibitors that have not met their post-marketing requirements demonstrating confirmatory benefit. This included a meeting of the Oncologic Drugs Advisory Committee in April and subsequent discussion with the FDA.

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Treatment Trials Therapies Disease 52

The Pharma Data

MAY 18, 2021

5 percent of the tumour area) as determined by an FDA-approved test or are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. About bladder cancer In 2020, there were over half a million new cases of bladder cancer diagnosed globally, with around 212,500 deaths from the disease.

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FDA Treatment Pharmaceuticals Science 40

The Pharma Data

FEBRUARY 8, 2021

Based on these data, ADT alone should no longer be considered sufficient for patients with advanced, castration-sensitive disease.”. The recruitment period for the study spanned from December 2015 to July 2017. Monitor for signs and symptoms of ischemic heart disease. ERLEADA ® received U.S.

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The Pharma Data

MARCH 11, 2021

In 2017, Opdivo was granted accelerated approval by the FDA as a single agent for patients with HCC who have been previously treated with sorafenib. The accelerated approval was based on tumor responses from the Phase 1/2 CheckMate -040 trial.

Treatment 52

Treatment Trials Therapies FDA Approval 52

Practical Cheminformatics

AUGUST 3, 2023

Many papers I’ve read recently use the MoleculeNet dataset, released by the Pande group at Stanford in 2017, as the “standard” benchmark. The ability of a compound to enter the CNS can also be facilitated or hindered by certain disease states. This is a mistake. The molecules span the gamut from antibiotics to zinc oxide.

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Drugs Biochemical Assays Metabolic Stability Screening Hits 94

The Pharma Data

JUNE 18, 2021

“We are dedicated to helping more people living with rheumatoid arthritis reach their treatment goals aimed at remission or low disease activity,” said Mudra Kapoor, M.D., Low Disease Activity per DAS28-CRP?3.2 Low Disease Activity per CDAI?10 Low Disease Activity per CDAI is defined as CDAI less than or equal to 10.

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