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.–

Production 100

Production Licensing Licensing Treatment 100

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. With further development, it could pave the way for treatments that are administered only once and have fewer side effects.

Engineering 145

Engineering Packaging DNA Treatment 145

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.

Therapies 59

Therapies Treatment Engineering Molecular Biology 59

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.

Therapies 105

Therapies Engineering Molecular Biology Science 105

Drug Target Review

JULY 12, 2023

Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). How does the DCI mechanism compare to the design of other drugs for cancer treatment?

Biochemical Assays 98

Biochemical Assays Treatment Bioinformatics Disease 98

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.

Small Molecule 105

Small Molecule Therapies Regulations Treatment 105

Drug Target Review

OCTOBER 4, 2023

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. To overcome these obstacles, combination therapy, which delivers multiple small molecules, has emerged as a potential solution.

Small Molecule 126

Small Molecule Drugs Immune Response Targeted Protein Degradation 126

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.

Therapies 56

Therapies RNA Pharma Companies Disease 56

PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

Therapies 69

Therapies Immune Response DNA Virus 69

Drug Target Review

MARCH 3, 2025

The search for effective treatments for neurodegenerative diseases like Parkinson’s disease has long been hindered by the brain’s complexity and the absence of adequate models for drug discovery. This approach has the potential to revolutionise clinical trial design and lead to more effective, personalised treatments.

Disease 52

Disease Treatment Therapies Cell Biology 52

The Pharma Data

FEBRUARY 22, 2021

Efanesoctocog alfa, a novel and investigational factor VIII therapy independent of von Willebrand Factor, is designed to provide near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen. In 2019, Sobi’s revenues amounted to SEK 14.2

FDA 52

FDA Treatment Therapies International 52

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 52

The Pharma Data

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. In addition, health-related quality of life (HRQoL), per total Functional Assessment of Cancer Therapy–Prostate (FACT-P), continued to be maintained with ERLEADA ®.

Treatment 52

Treatment Pharmaceutical Companies Therapies Pharmaceuticals 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. and Celgene K.K.,

Therapies 52

Therapies Trials Treatment Disease 52

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. MIAMI, Jan. 25, 2021 (GLOBE NEWSWIRE) — Ambulero, Inc. ,

Therapies 52

Therapies Disease Licensing Licensing 52

The Pharma Data

JUNE 1, 2022

The United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A, a rare and life-threatening bleeding disorder, based on data from the pivotal XTEND-1 P hase 3 stud y.

Therapies 52

Therapies FDA Science Treatment 52

Broad Institute

NOVEMBER 13, 2023

This commitment to the Gerstner Center for Cancer Diagnostics includes $10 million for current support for the Center and $10 million in endowed funding, adding to the $15 million originally committed by Gerstner and an initial endowment of $10 million from the Eli & Edythe Broad Foundation to establish the Center in 2019.

DNA 116

DNA Medical Schools Treatment Therapies 116

The Pharma Data

JUNE 2, 2023

Phase 3 Studies of Pfizer’s Novel Antibiotic Combination Offer New Treatment Hope for Patients with Multidrug-Resistant Infections and Limited Treatment Options Pfizer Inc. No patient treated with ATM-AVI experienced a treatment-related SAE. million deaths globally were caused by bacterial AMR in 2019 alone.

Treatment 52

Treatment Hospitals International Therapies 52

The Pharma Data

MARCH 2, 2021

Boehringer Ingelheim and Gubra today announced a new research and licensing agreement focused on the identification and validation of targets and innovative peptide compounds for the treatment of obesity. iii Current treatment options have limited efficacy in terms of weight loss or are often associated with adverse events. “We

Treatment 52

Treatment Bioinformatics Clinical Development Therapies 52

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 Engineering Clinical Development 52

The Pharma Data

DECEMBER 17, 2020

18, 2020 /PRNewswire/ — Camurus AB (NASDAQ STO: CAMX) announced today that the Swiss agency for therapeutic products, Swissmedic, has approved weekly and monthly Buvidal ® prolonged release buprenorphine for the treatment of opioid dependence in adults and adolescents from 16 years of age. LUND, Sweden , Dec. About Buvidal.

Treatment 52

Treatment Clinical Trials International Pharmaceutical Companies 52

Broad Institute

JUNE 21, 2023

Now, researchers at the Broad Institute of MIT and Harvard have found that treatment with continuous hypoxia — low-oxygen conditions comparable to levels at a Mount Everest base camp — restores balance and coordination in a mouse model of Friedreich’s ataxia. A few treatments stood out.

Treatment 98

Treatment Disease Molecular Biology Medical Schools 98

Drug Target Review

OCTOBER 26, 2023

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Molecular Therapy 20 , 1831-1832 (2012). e419 (2023).

Engineering 105

Engineering Virus Therapies Immune Response 105

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.

FDA Approval 52

FDA Approval Therapies FDA Hospitals 52

Drug Target Review

AUGUST 24, 2023

We will explore how GDF-15 impacts the tumour microenvironment and hinders the infiltration of T cells into the tumour, as well as the implications of neutralising GDF-15 to reverse its inhibitory effects and sensitise tumours to anti-PD-1 treatment. T-cell infiltration is a prerequisite for responses to checkpoint inhibitors.

Treatment 98

Treatment Therapies Clinical Development Cell Biology 98

The Pharma Data

JANUARY 12, 2021

In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies. Photo courtesy of Bayer.

Therapies 52

Therapies Licensing Licensing Pharmaceuticals 52

The Pharma Data

SEPTEMBER 27, 2021

AbbVie Submits Regulatory Application to FDA for Risankizumab-rzaa (SKYRIZI®) for the Treatment of Patients 16 Years and Older with Moderate to Severe regional enteritis. The ADVANCE study included a mixed population of patients who had responded inadequately or are intolerant to standard and/or biologic therapy.

Treatment 52

Treatment FDA Therapies Disease 52

Drug Target Review

DECEMBER 4, 2023

Tailoring medical care to a patient’s unique genetic makeup, alongside the close analysis of disease progression, leads to more effective treatments, reduced side effects and faster diagnoses. What’s more, it remains an open question as to whether our healthcare facilities are currently equipped to deliver such treatment programmes at scale.

Treatment 105

Treatment Science DNA Doctors 105

The Pharma Data

JUNE 28, 2021

“Despite available treatments, many people with moderate to severe forms of this disease continue to experience a relentless and burdensome cycle of skin and itch symptoms. . We are encouraged that the CHMP has recognized RINVOQ’s potential as an additional treatment option for these patients.”

Treatment 52

Treatment Therapies Disease Vaccine 52

The Pharma Data

MAY 16, 2022

Food and Drug Administration (FDA) has lifted the clinical hold placed on the company’s Investigational New Drug Application (IND) to evaluate injectable lenacapavir for HIV treatment and HIV pre-exposure prophylaxis (PrEP). There is no cure for HIV or AIDS. About Lenacapavir.

Treatment 52

Treatment FDA Science Therapies 52

The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

Disease 52

Disease Trials Treatment Clinical Trials 52

The Pharma Data

DECEMBER 2, 2020

(NYSE: PFE) today announced that the first participant has been dosed in the Phase 3 BASIS study of marstacimab (PF-06741086), an anti-tissue factor pathway inhibitor (anti-TFPI) being evaluated for the treatment of people with severe hemophilia A or B, with or without inhibitors. About the BASIS study.

Therapies 52

Therapies Treatment Disease Clinical Trials 52

The Pharma Data

MARCH 9, 2022

The key secondary endpoint was also met, demonstrating once-weekly efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy, showing a statistically significant reduction in ABR based on intra-patient comparison. Efanesoctocog alfa was well-tolerated, and inhibitor development to factor VIII was not detected.

Treatment 52

Treatment Pharmacokinetics Therapies FDA 52

Drug Target Review

APRIL 26, 2023

1 RNA-delivery particles have the potential to revolutionise the treatment of lung diseases such as cystic fibrosis. The researchers hope that this new technology could lead to the development of more effective treatments for these conditions, which can be debilitating and often life-threatening. References: 1. What is genome editing?

RNA 98

RNA Medical Schools Engineering Disease 98

Eye on FDA

JULY 5, 2022

Over the years, Congress has acted to enhance the ability to bring more new treatments from test tube to medicine chest more quickly. Most notably in 2012 the Food and Drug Administration Safety and Innovation Act (FDASIA) was signed into law, bring to bear among other things, breakthrough therapy designation. The news is not so hot.

Clinical Trials 81

Clinical Trials FDA Therapies Treatment 81

The Pharma Data

MARCH 9, 2021

“While results from the CANOPY-2 trial are not what we hoped for in patients with advanced or metastatic non-small cell lung cancer who have been treated with other lines of therapy, these data give us valuable insights into IL-1? The adjuvant study is designed to determine if treatment with canakinumab can prevent cancer relapse 3.

Treatment 52

Treatment Immune Response Trials Clinical Trials 52

Drug Target Review

SEPTEMBER 4, 2023

7,9 Dihydrofolate reductase-thymidylate synthase (DHFR) have been successfully exploited for the therapy of bacterial infections and other parasitic diseases such as malaria. 2019): CRISPR in Parasitology: Not Exactly Cut and Dried! 2019): Biological plasticity rescues target activity in CRISPR knockouts, Nature Methods.

Drugs 111

Drugs RNA DNA Disease 111