New Drug Approvals

APRIL 25, 2025

3] Primary endocrine resistance was defined as relapse while on the first two years of adjuvant endocrine therapy (ET) and secondary endocrine resistance was defined as relapse while on adjuvant ET after at least two years or relapse within twelve months of completing adjuvant ET. [3] New Drug Therapy Approvals 2024 (PDF). 20 May 2019.

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DrugBank

JUNE 18, 2025

Before a therapy can be approved for patient use, it must undergo extensive clinical testing and strictly adhere to regulatory guidelines. For example, the FDA’s Breakthrough Therapy Designation grants priority review to medicines demonstrating substantial improvement over existing treatments.

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Drug Target Review

MARCH 3, 2025

In this interview, Professor Schwamborn discusses his research, the promise of brain organoids, and his vision for the future of stem cell-based therapies. ” By using personalised models, researchers can identify subgroups of patients with similar molecular characteristics, potentially leading to the development of targeted therapies.

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New Drug Approvals

MAY 17, 2025

Jump up to: a b New Drug Therapy Approvals 2023 (PDF). Liu A (1 May 2019). Archived from the original on 25 April 2023. Retrieved 25 April 2023 via GlobeNewswire. Food and Drug Administration (FDA) (Report). January 2024. Archived from the original on 10 January 2024. Retrieved 9 January 2024. ^

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The Pharma Data

JUNE 15, 2025

This update comes after two cases of fatal acute liver failure (ALF) were reported in non-ambulatory DMD patients who received Elevidys, a phenomenon that underscores the ongoing complexities and safety signals related to adeno-associated virus (AAV)-mediated gene therapy.

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Codon

JUNE 5, 2025

.” Four years earlier, in 2019, Gray had become the first patient with sickle cell anemia — a genetic disorder that causes red blood cells to become sticky and rigid — to receive an experimental treatment using CRISPR genome editing.

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Broad Institute

FEBRUARY 26, 2025

These gene signatures provide a roadmap that the field can use to study myeloid cells and how they impact the way brain tumors respond to therapy, Bernstein said. Bradley Bernstein , an institute member at the Broad and chair of the cancer biology department at DFCI, was the studys senior author.

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New Drug Approvals

APRIL 18, 2025

2] The US Food and Drug Administration (FDA) granted the application for crinecerfont fast track , breakthrough therapy , orphan drug , and priority review designations. [2] New Drug Therapy Approvals 2024 (PDF). World Health Organization (2019). 2] The FDA granted the approval of Crenessity to Neurocrine Biosciences, Inc. [2]

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FDA FDA Approval Drugs Trials 62

New Drug Approvals

APRIL 19, 2025

New Drug Therapy Approvals 2024 (PDF). Archived from the original (PDF) on 16 June 2019. “Metoprolol Therapy and CYP2D6 Genotype” In Pratt VM, McLeod HL, Rubinstein WS, et al. Yamashita T, Nakasu Y, Mizutani H, Sumitani K (2019). A further publication disclosing it is US5013734. 1 October 2024. January 2025.

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BioPharma Drive: Drug Pricing

JUNE 26, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime.

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Licensing Licensing Therapies Vaccine 117

Codon

JULY 21, 2025

For others, like gene therapy, focusing on nucleotides and amino acids holds more promise. They got very good at StarCraft 2 up to 2019, but still haven’t transcended the best human players. Perhaps “biology” is too big of a tent to expect that everyone inside will benefit from the same toolkit.

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BioPharma Drive: Drug Pricing

AUGUST 28, 2023

The treatment, developed by a biotech Bayer acquired in 2019, appeared safe in a small trial and showed signs of an effect on disease progression.

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New Drug Approvals

JULY 18, 2025

1] A phase II study (NCT04109183) was finished in March 2019 by Genuine Biotech. The patent was voided in 2019 after Riboscience, its new holder, stopped paying fees. [25] “Antiviral activity of FNC, 2′-deoxy-2′-β-fluoro-4′-azidocytidine, against human and duck HBV replication” Antiviral Therapy.

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RNA Clinical Trials Trials Virus 62

Drug Target Review

NOVEMBER 8, 2023

Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.

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Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. developing immune stem cell therapies for treating ovarian and gastric cancer.

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Therapies Clinical Trials Cell Biology Trials 118

PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

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Therapies FDA Clinical Trials Disease 101

Broad Institute

JULY 19, 2023

Researchers reprogram gene therapy viral vectors to bind specific protein targets By Allessandra DiCorato July 19, 2023 Breadcrumb Home Researchers reprogram gene therapy viral vectors to bind specific protein targets A new screening method zeroes in on adeno-associated viruses that enter the brain through a defined mechanism.

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Drug Target Review

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

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Therapies Engineering Molecular Biology Science 105

Broad Institute

JULY 10, 2024

A three-drug cocktail known as Trikafta has greatly improved patient quality of life since its development in 2019, but can cause cataracts and liver damage and must be taken daily at a cost of about $300,000 per year. The most common is a three base-pair CTT deletion that causes the ion channel protein to misfold and degrade.

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Antidote

JULY 29, 2022

billion to bring a new therapy to market. In 2019 alone, research and development spending totaled $186 billion for the global pharmaceutical industry. Between 2009 and 2018, U.S.

Clinical Trials 96

Clinical Trials Trials Therapies Pharmaceuticals 96

Drug Discovery Today

DECEMBER 17, 2019

December 10, 2019 — Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, gene therapies, and consumer health products, today announced that it has completed clinical production of Bridge Therapeutics Inc.’s Somerset, N.J. Birmingham, Ala.–

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? 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.

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Therapies Treatment Engineering Molecular Biology 59

The Pharma Data

JANUARY 19, 2021

Estrogen Therapy Need Not Stop for Gender-Affirming Surgery. 19, 2021 — For transgender and gender nonbinary (TGNB) patients undergoing primary vaginoplasty, perioperative venous thromboembolism (VTE) occurs rarely, regardless of whether hormone therapy is suspended prior to surgery, according to a study published online Jan.

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Therapies International Research 52

Drug Target Review

JULY 11, 2024

To date, there are less than 30 known surface proteins that form the basis of all approved cancer-targeted therapies of all modalities, including antibody-drug conjugates (ADCs), T-cell engagers, CAR T-cells and radiopharmaceuticals. These could be targets for antibody-drug conjugates (ADCs), bi-specific antibodies, or other therapies.

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Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.

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The Pharma Data

JANUARY 25, 2021

a biotechnology company developing cell and gene therapy treatments for patients suffering from vascular disease, today announced that it raised up to $5.5M that will lead clinical testing of a novel gene therapy for a serious vascular disease in Europe. The company is a 2019 spin-out of the University of Miami co-founded by Robert L.

Therapies 52

Therapies Disease Licensing Licensing 52

Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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The Pharma Data

JUNE 2, 2022

New multi-program collaboration to develop allogeneic TCR-T/CAR-T programs brings together Immatics’ allogeneic gamma delta T cell therapy platform ACTallo ® with Bristol Myers Squibb’s technologies and oncology drug development expertise. Chief Executive Officer and Co-Founder of Immatics. “We FRCP, D.Phil.,

Therapies 52

Therapies Production Clinical Development Engineering 52

The Pharma Data

MARCH 25, 2021

this week announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved Breyanzi (lisocabtagene maraleucel: liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy for the treatment of patients with relapsed or refractory (R/R) large B-cell lymphoma 1 and R/R follicular lymphoma. Bristol Myers Squibb K.K.

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Therapies Trials Treatment Disease 52

The Pharma Data

AUGUST 4, 2020

Therapy has been approved to treat advanced NTRK fusion-positive solid tumours. The European Commission (EC) has granted conditional marketing authorisation for Rozlytrek (entrectinib) for the treatment of advanced solid tumours expressing a neurotrophic tyrosine receptor kinase (NTRK) gene fusion.

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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The Pharma Data

NOVEMBER 1, 2020

Corlieve’s lead program employs a novel AAV gene therapy approach for the treatment of refractory temporal lobe epilepsy (TLE), the most commonly diagnosed focal epilepsy in humans. Maryland , US), a leading gene therapy company. Maryland , US), a leading gene therapy company. PARIS , Nov.

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Therapies Science Treatment Disease 52

The Pharma Data

APRIL 19, 2022

Kite’s Global CAR T-Cell Therapy Manufacturing Network Increasing Capacity by 50% to Meet Patient Demand for New Cancer Therapies. — Scalable and Adaptable Facility Provides Flexibility for Current and Future Cell Therapy Innovation. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.

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The Pharma Data

DECEMBER 21, 2020

The FDA granted cilta-cel Breakthrough Therapy and Orphan Drug designations in 2019, and the therapy received a PRIME Priority Medicines designation from the European Commission (EC) that same year and an Orphan Drug designation from the EC in February 2020. Source link.

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The Pharma Data

OCTOBER 21, 2020

In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days. Source: GSK .

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Clinical Development Therapies Clinical Trials Treatment 52

Drug Target Review

DECEMBER 12, 2023

He was the founding CEO since the inception of Araris in 2019 securing $40M in financing from Swiss, UK, US and Korean investors and $2.5M He positioned Araris to shift the paradigm in how to think about developing targeted cancer therapies and since October 2023, he transitioned to become its CSO.

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DrugBank

OCTOBER 4, 2024

The FDA's accelerated approval pathways, such as the Breakthrough Therapy designation and the Priority Review program, have shortened development timelines and reduced costs for certain drugs, making them more attractive acquisition targets. However, such acquisitions also entail significant risks for biotech firms.

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