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. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Molecular Therapy 20 , 1831-1832 (2012). Nature 578 , 229-236 (2020). e419 (2023).

Engineering 105

Engineering Virus Therapies Immune Response 105

Drug Target Review

NOVEMBER 6, 2023

2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).

Small Molecule 111

Small Molecule Therapies Treatment Disease 111

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.

Disease 52

Disease Treatment Therapies Cell Biology 52

DrugBank

AUGUST 15, 2024

To effectively navigate this ecosystem and expedite the development of new therapies, collaboration between the pharmaceutical industry and academia is proving increasingly vital. An example is the collaboration between Novartis and the University of Oxford to develop a gene therapy for spinal muscular atrophy, a rare genetic disease.

Drug Development 98

Drug Development Clinical Trials Drugs Therapies 98

The Pharma Data

JANUARY 4, 2021

was granted with the “Breakthrough Therapy Designation Drug” by the Center for Drug Evaluation (CDE) and currently enter a phase III clinical trial for treatment of patients with small cell lung cancer (SCLC) with disease progression or recurrence after two lines of prior systematic chemotherapy. SHENZHEN, China , Jan.

Therapies 40

Therapies Clinical Trials Drug Development Drugs 40

The Pharma Data

APRIL 22, 2023

Alarmingly, since 2013, the world-wide prevalence of MS has risen by 30%. While no cure is available for MS, existing disease-modifying therapies in the form of small molecule and protein drugs either directly target the self-reactive immune cells or broadly dampen inflammation. who is a Core Faculty member at the Wyss Institute.

Therapies 40

Therapies Immune Response Small Molecule Engineering 40

The Pharma Data

DECEMBER 22, 2020

Primary endpoints of this study include safety, tolerability, and tumor response of the ASP-1929 photoimmunotherapy in combination with anti-PD1 therapy. Outside of Japan , Illuminox therapies have not yet been approved as safe or effective by any regulatory authority. Since 2013, Rakuten Medical, Inc. SAN MATEO, Calif. ,

Clinical Trials 52

Clinical Trials Therapies Trials Production 52

Drug Target Review

JUNE 12, 2023

TEAD proteins are known to be very important in cancer progression TEAD proteins are known to be very important in cancer progression, and there are a number of therapies in development. Can you provide a summary of the key findings and implications of the preclinical studies on ISM6331 for the treatment of advanced solid tumours?

Metabolic Stability 92

Metabolic Stability Clinical Trials Small Molecule Pharmacokinetics 92

LifeSciVC

OCTOBER 13, 2022

This summer, approvals on both sides of the Atlantic – for beta thalassemia and cerebral adrenoleukodystrophy in the United States and for severe hemophilia A and aromatic L-amino acid carboxylase deficiency in Europe – show that regulators are likely open to green-lighting a range of emerging gene therapies.

Therapies 40

Therapies Disease Treatment Marketing 40

Codon

JULY 14, 2024

While this departs from the reductionist, single-component therapies that have dominated transfusion medicine since World War II, emerging data underscores that whole blood transfusions—blood with all its parts—yield better outcomes following severe blood loss than transfusions involving discrete blood components. Always free.

Production 140

Production Clinical Trials Trials Therapies 140

The Pharma Data

OCTOBER 23, 2020

breast cancer units between 2013 and 2018. The propensity-matched analysis included 426 surgery and 240 primary endocrine therapy (PET) patients matched for age, fitness, and frailty. Lynda Wyld, M.B.Ch.B., The researchers found that the median age varied by treatment allocation (surgery: 76 years; PET: 84 years). © 2020 HealthDay.

Therapies 52

Therapies Treatment Research 52

Eye on FDA

MARCH 7, 2024

FDA currently has over 30 advisory committees providing the agency with access to advice from experts outside of government to help in evaluating new therapies for safety and efficacy as well as defining policy positions around many of the principles that guide such decisions.

FDA 78

FDA Disease Government Drugs 78

The Pharma Data

SEPTEMBER 7, 2020

Novartis will present 48 abstracts from its leading MS portfolio, including new data on recently FDA-approved Kesimpta ® (ofatumumab) — the first and only self-administered, targeted B-cell therapy for relapsing forms of MS (RMS )— Mayzent ® (siponimod) and Gilenya ® (fingolimod) . Atlas of MS 2013. Adv Studies Pharm. Types of MS.

Therapies 52

Therapies Treatment Clinical Trials Disease 52

Drug Target Review

APRIL 4, 2024

The animal model is based on creating a dopamine-deficient state, by a specific lesion of dopaminergic cells, and then exposing it to repeated substitution therapy with L-DOPA. In 2013, Nicholas co-founded IRLAB Sweden. This creates a condition which mimics the state in advanced PD in humans.

Disease 105

Disease Treatment Licensing Licensing 105

PPD

SEPTEMBER 25, 2023

Overall, since 2013, the total number of trial objectives, endpoints and eligibility criteria has grown across all clinical trial phases, likely driven in part by the robust growth of oncology trials. In Asia , personalized and precision medicine has become the driving force behind transformative clinical trials.

Drug Development 104

Drug Development Clinical Trials Drugs Trials 104

The Pharma Data

DECEMBER 8, 2020

MRI-guided laser interstitial thermal therapy, a minimally invasive procedure for kids who have drug-resistant epilepsy, is successful in more than half of all cases and has a short recovery time, researchers report. The researchers reported on outcomes of 182 children who received the therapy in the past seven years.

Hospitals 52

Hospitals Therapies Doctors Research 52

The Pharma Data

DECEMBER 30, 2020

She’s a professor of physical therapy at Washington University in St. Before the study began in December 2013, all participants had struggled with lower back pain for at least a year. Louis School of Medicine. Lower back pain is incredibly common, and the No. 1 cause of disability, according to Van Dillen.

Therapies 52

Therapies Treatment Doctors Research 52

The Pharma Data

JUNE 18, 2021

The approval confirms Aubagio as the first oral multiple sclerosis (MS) therapy for first-line treatment of children and adolescents with MS in the European Union. The EC approval is based on data from the Phase 3 TERIKIDS study. MS affects an estimated 2.8 Therapeutic Area Head, Neurology Development at Sanofi Genzyme. “The

Treatment 40

Treatment Therapies Disease Trials 40

Drug Target Review

SEPTEMBER 28, 2023

Twist was officially founded in 2013. Once a specific mutation is detected, clinicians can look through available precision medicine therapies to find one to treat the disease or develop a new personalised therapy to treat a patient’s specific form of disease, such as cancer. At that time synesthetic biology wasn’t cool.

DNA 116

DNA Organic Chemistry Science Virus 116

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. 2013): Exploring the Trypanosoma brucei Hsp83 Potential as a Target for Structure Guided Drug Design, PLOS Neglected Tropical disease. Trends in Parasitology. (35):

Drugs 111

Drugs RNA DNA Disease 111

The Pharma Data

MARCH 28, 2022

While these were not the results we were hoping for, they are clear and will inform future research across our broad pipeline of investigational ALS therapies. The company has continued to invest in and pioneer research despite making the difficult decision to discontinue a late-stage ALS asset in 2013. Biogen Inc.

RNA 52

RNA Therapies Disease Biosimilars 52

The Pharma Data

NOVEMBER 18, 2021

Senior Vice President, Early Clinical Development, Hematology/ Oncology and Cell Therapy and San Francisco point head. “ A private,non-profit institution, LabCentral was innovated in 2013 as a helipad for high implicit life- lores and biotech startups. Since opening in 2013, MBC BioLabs has helped launch and grow 230 companies.

Molecular Biology 52

Molecular Biology Pharmaceuticals Laboratories Clinical Development 52

The Pharma Data

MARCH 25, 2021

Many of these patients have exhausted available therapies, have not had a new class of medications available to them in more than a decade and are eager for new, non-opioid options,” said Ken Verburg, tanezumab development team leader, Pfizer Global Product Development. “The About the Pfizer-Lilly Alliance.

Treatment 52

Treatment FDA Therapies Licensing 52

The Pharma Data

MARCH 25, 2021

Despite its far-reaching impact, many patients still live with debilitating pain and have exhausted or are unable to take or tolerate currently available therapies. Despite its far-reaching impact, many patients still live with debilitating pain and have exhausted or are unable to take or tolerate currently available therapies.

Treatment 52

Treatment Therapies FDA Licensing 52

The Pharma Data

NOVEMBER 26, 2020

Constrained logistics of moving critical medical supplies have been a common theme across recent epidemics such as Ebola in 2013 to 2016, and now COVID-19. From the outset both outbreaks saw shortages of personal protective equipment and cleaning supplies, with particularly dire consequences for the frontline healthcare workers.

Drugs 52

Drugs Vaccine International Therapies 52

Drug Target Review

AUGUST 9, 2023

ALS Therapy Development Institute, www.als.net/news/frontotemporal-dementia-vs-als/. From Basic Research to the Clinic: Innovative Therapies for ALS and FTD in the Pipeline.” 79,3 (2013): 416-38. “Clinical and demographic factors and outcome of amyotrophic lateral sclerosis in relation to population ancestral origin”.

RNA 98

RNA Regulations Disease DNA 98

The Pharma Data

DECEMBER 21, 2020

Tagrisso was recently granted Breakthrough Therapy Designation for patients in the early-stage disease setting by the US FDA. 2013:6;2800-12. 2013:66;79-89. At two years, 89% of patients treated with Tagrisso remained alive and disease free versus 52% on placebo after surgery, the current standard of care. Int J Clin Exp Pathol.

Treatment 52

Treatment Trials Disease FDA 52

FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

An Opportunity to Convene this New Advisory Committee for All Rare Disease Therapies In 2018, HP&M attorneys Frank Sasinowski and James Valentine proposed a Rare Disease Center of Excellence (which we blogged about here ).

Disease 59

Disease Production FDA Drug Development 59

Drug Target Review

SEPTEMBER 26, 2023

Early detection of neurotoxicity induced by potential new therapies is a major challenge, and hiPSC-neuronal cells may provide a solution. This shift occurred following implementation of the Comprehensive in Vitro Proarrhythmia Assay (CiPA) initiative in 2013 and extensive testing of hiPSC-cardiomyocytes with known proarrhythmic drugs.

Drugs 111

Drugs FDA Disease Animal Testing 111

The Pharma Data

APRIL 17, 2021

TECFIDERA was approved under the National Medical Products Administration priority review process evaluating therapies with urgent clinical needs. First introduced in 2013, TECFIDERA has demonstrated a well-established safety and efficacy profile with more than 10 years of data from clinical trials and real-world experience.

Treatment 52

Treatment Clinical Trials Disease Trials 52

The Pharma Data

NOVEMBER 3, 2020

m (-), including US launch of the scientifically proven digital therapies deprexis® and vorvida®. These changes in prioritization have been made to ensure we have all the resources needed to secure a successful launch of the digital therapies and to ensure OX124 development is finalized and meet FDA’s requirement.

Therapies 52

Therapies Pharmaceuticals Production Marketing 52

The Pharma Data

JANUARY 13, 2021

Lancet Respir Med 2013. In addition, nebulized ensifentrine showed further improved lung function and reduced lung volumes in COPD patients taking standard short- and long-acting bronchodilator therapy, including maximum bronchodilator treatment with dual/triple therapy. Franciosi LG, et al., . .

Hospitals 52

Hospitals Clinical Trials Therapies Treatment 52

The Pharma Data

JANUARY 7, 2021

RMI), a global biotechnology company developing precision-targeted cancer therapies based on its proprietary, anti-cancer treatment platform, Illuminox , will present at the 39th Annual J.P. Outside of Japan, Illuminox therapies have not yet been approved as safe or effective by any regulatory authority. SAN MATEO, Calif. ,

Clinical Trials 40

Clinical Trials Therapies Trials Licensing 40

Elrig

JUNE 18, 2024

Supporting yourself to be compassionate and understanding will help those around you too (and often make you a better researcher too) Question 2 – What measures can be put in place to make therapy more accessible to doctoral and early career researchers?

Science 52

Science Therapies RNA Research 52

Drug Target Review

APRIL 8, 2024

How have recent advancements in immune checkpoint blockade therapy affected outcomes for primary NSCLC? Dr Govindan : Even though immunotherapy and chemotherapy given after surgery improve the overall survival, as Dr Cote said, many may not need those therapies and a good number of them recur despite post operative systemic therapies.

Hospitals 52

Hospitals Therapies Clinical Trials Molecular Biology 52

The Pharma Data

SEPTEMBER 15, 2020

Amgen is a proud sponsor of MBC BioLabs, assisting high-potential and innovative early-stage life science and biotech companies accelerating development of new therapies to improve human health. We look forward to interacting with Amgen ‘s scientific and therapeutic experts to help move Trilomer® therapies into the clinic.

Targeted Protein Degradation 52

Targeted Protein Degradation Molecular Biology Science Small Molecule 52

The Pharma Data

MARCH 23, 2022

Dr. Baynes joined Merck in 2013. Dr. Barr oversaw the company’s Vaccines/Infectious Disease area during a period of high productivity, including the development of novel therapies for chronic hepatitis C and HIV-1 infections. I know there are many who join me in thanking Roy for his efforts and wishing him well in his next chapter.”.

Clinical Development 52

Clinical Development Vaccine Research Laboratories Laboratories 52