Drug Discovery Today

NOVEMBER 5, 2020

London, November 4th, 2020: NanoMab Technology Limited, a privately held biopharmaceutical company focussing on cancer precision therapies, today announced that it had received CTA Acceptance from the Medicines Healthcare products Regulatory Agency (MHRA) to carry out a Phase II clinical Study for its NM-01 product.

Production 100

Production Therapies 100

Drug Target Review

MARCH 21, 2024

There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. AAV-based therapies often can’t be dosed more than once due to concerns about an immune response. 2020 Jul 3;10(1):10967. Molecular Therapy-Nucleic Acids.

Therapies 64

Therapies RNA Immune Response Treatment 64

Drug Target Review

SEPTEMBER 6, 2024

What trends are driving the increased use of in vivo delivery methods in gene therapy? Being a specialist lentiviral vector CDMO, at VIVEbiotech, this annual growth rate has been higher with a CAGR from 2020 to 2023 of almost a 50 percent. How are new therapeutic areas emerging as potential targets for in vivo gene therapy?

Therapies 59

Therapies Vaccine Marketing Disease 59

Eye on FDA

AUGUST 18, 2020

But it is not likely to surprise anyone that the first half of 2020 has caused not only an increase in communication from FDA, but a focus as well. Approvals – There were 54 press statements regarding approvals so far in 2020, comparing to 42 approvals announced in 2019.

FDA 61

FDA Production Drugs Therapies 61

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Therapies 52

Therapies Clinical Trials FDA Production 52

SugarCone Biotech

MARCH 24, 2025

When we look at how TCEs interact with targeted cancer cells we can quickly see how cancers respond to T Cell-based therapies. The most common response is quite expected, which is that the cancer cells escape from TCE therapy by downregulating expression of the targeted cancer protein (Ref. Proc Natl Acad Sci U S A 115: E2068-E2076.

Immune Response 64

Immune Response Therapies Treatment Disease 64

Drug Target Review

NOVEMBER 7, 2023

To reach the other 40 percent of those proteins, Stanford ChEM-H researchers pioneered lysosome targeting chimeras (LYTACs) in 2020. To test which patients are more likely to respond to LYTAC therapy, the level of neddylated CUL3 could be measured. Enzyme replacement therapy is a frequent treatment for these disorders.

Therapies 59

Therapies Science Disease Treatment 59

Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% between 2020-2027.

Therapies 52

Therapies Research Clinical Trials Trials 52

Codon

NOVEMBER 3, 2024

The experience showed me that new therapies are needed not only to meet the targets laid out by the End TB Strategy but also to prevent drug resistance from negating the effectiveness of current therapeutics. Cell (2020). coli plated onto media containing halicin or ciprofloxacin antibiotics, after 7 days of incubation.

Vaccine 100

Vaccine Trials Therapies Treatment 100

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

Engineering 105

Engineering Virus Therapies Immune Response 105

The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

Production 52

Production Therapies DNA Virus 52

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

The Pharma Data

AUGUST 5, 2021

The acquisition strengthens BioNTech’s cell therapy pipeline by accelerating the individualized solid tumor Neoantigen TCR cell therapy research and development program. It also expands the Company’s cell therapy capabilities and manufacturing footprint in North America, building on its acquisition of Neon Therapeutics in 2020.

Therapies 52

Therapies Small Molecule Vaccine Science 52

PPD

DECEMBER 16, 2024

Internal costs: The complexity of protocol designs a necessity for innovative therapies often requires more diverse patient populations, more extensive data collection and sophisticated trial methodologies, all of which demand higher financial outlays. Nearly 39% of sponsors cite these costs as primarily driven by complex protocols.

Drug Development 52

Drug Development Clinical Trials Compliance Trials 52

The Pharma Data

FEBRUARY 4, 2021

3, 2021 /PRNewswire/ — AbbVie (NYSE: ABBV) announced financial results for the fourth quarter and full year ended December 31, 2020. AbbVie hosted an Immunology Strategic Update event on December 14, 2020 for members of the investment community. NORTH CHICAGO, Ill., Gonzalez , chairman and chief executive officer, AbbVie.

Production 52

Production Clinical Trials International Small Molecule 52

The Pharma Data

JANUARY 25, 2021

Nasdaq: ALXN) today announced that the Company will report its financial results for the fourth quarter and full year ended December 31, 2020 before the US financial markets open on February 4, 2021. BOSTON –(BUSINESS WIRE)–Jan. 25, 2021– Alexion Pharmaceuticals, Inc. AstraZeneca and Alexion Combination.

Production 52

Production Science Pharmaceuticals Marketing 52

The Pharma Data

SEPTEMBER 11, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. o Completion: 2020. o Number of patients: 3,730.

Therapies 52

Therapies Hospitals Clinical Trials Trials 52

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects.

Small Molecule 145

Small Molecule Drugs Clinical Pharmacology Trials 145

Drug Target Review

SEPTEMBER 22, 2023

Organoid technologies are becoming an invaluable solution for preclinical research, with the ability to augment the development of personalised medicine, drug discovery and gene therapies. Organoids for cell therapy and drug discovery. Elsevier; 2020. 2020 Oct 7;21(10):571–84. 2020 Jun 8;4(9):863–74.

Clinical Research 116

Clinical Research Research Drug Development Molecular Biology 116

The Pharma Data

SEPTEMBER 27, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with coronary failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. 2,3 a number one explanation for hospitalization within the U.S.

Therapies 52

Therapies Clinical Trials Hospitals Trials 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.

Therapies 52

Therapies Trials Treatment Disease 52

DrugBank

AUGUST 9, 2024

These new therapies have demonstrated significant LDL cholesterol reductions, but their high cost remains a barrier to widespread adoption. While research into disease-modifying therapies is ongoing, developing such treatments remains complex and challenging, requiring a sustained commitment from academia and industry.

Pharmaceuticals 98

Pharmaceuticals Research Pharmaceutical Companies Clinical Trials 98

DS in Pharmatics

JUNE 10, 2023

On April 12, 2023, BeNeLuxA1 announced that negotiations failed to secure reimbursement for Libmeldy, a gene therapy developed by Orchard Therapeutics for the treatment of metachromatic leukodystrophy, on grounds of price. In the UK, an initial application to NICE was rejected as well back in 2020. This is a huge […]

Marketing 66

Marketing Therapies Treatment 66

The Pharma Data

APRIL 7, 2022

This unprecedented CMS decision effectively denies all Medicare beneficiaries access to ADUHELM ® (aducanumab-avwa), the first and only FDA-approved therapy in a new class of Alzheimer’s drugs. In 2020, Biogen launched a bold 20-year, $250 million initiative to address the deeply interrelated issues of climate, health, and equity.

Treatment 52

Treatment Therapies Disease FDA Approval 52

The Pharma Data

APRIL 27, 2022

Based on DESTINY-Breast04 results where AstraZeneca and Daiichi Sankyo’s Enhertu demonstrated a significant improvement in both progression-free survival and overall survival Enhertu has now been granted five Breakthrough Therapy Designations, including three in breast cancer and one in both lung and gastric cancers.

Therapies 52

Therapies Trials Protein Expression Treatment 52

The Pharma Data

MAY 27, 2022

of patients with RRMM responding to therapy (98 percent overall response rate ORR ).1,6 “The approval of Janssen’s first cell therapy is testament to our ongoing commitment to advance science and transform outcomes for those living with multiple myeloma,” said Sen Zhuang, M.D., In December 2017, Janssen Biotech, Inc.

Therapies 52

Therapies Treatment Pharmaceutical Companies Nurses 52

The Pharma Data

APRIL 22, 2023

The Atlas of MS reported in 2020 that someone is diagnosed with MS every five minutes around the world, adding to about 2.8 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.

Therapies 40

Therapies Immune Response Small Molecule Engineering 40

DrugBank

OCTOBER 4, 2024

For example, Gilead Sciences' acquisition of Immunomedics in 2020 included Trodelvy, an antibody-drug conjugate approved for treating triple-negative breast cancer. The $63 billion acquisition of Allergan by AbbVie in 2020 stands as a prime example. However, such acquisitions also entail significant risks for biotech firms.

Pharmaceuticals 98

Pharmaceuticals Pharmaceutical Companies Therapies Marketing 98

Drug Target Review

OCTOBER 30, 2024

Organoids will play increasingly crucial roles not only in research and diagnostics, but also in therapies to restore and enhance the human body. MedComm (2020). Aside from kidney repair, some other areas of active research include the transplantation of organoids to repair retinal degeneration and to repair colon injury.

Treatment 105

Treatment Disease Clinical Trials Research 105

Creative Biolabs

MAY 25, 2022

Cancer has become the second largest health threat in the world, with about 10 million people dying of cancer in 2020. For decades, cytotoxic-based chemotherapy has been the main treatment for all kinds of cancers. These cytotoxic drugs include DNA base analogs, antimetabolic drugs, tubulin inhibitors, etc.

Therapies 52

Therapies Drugs DNA Treatment 52

Drug Target Review

OCTOBER 4, 2023

Dual payload ADCs As effective as therapies have been in treating solid and haematological cancers, tumour heterogeneity and resistance remain major clinical challenges. To overcome these obstacles, combination therapy, which delivers multiple small molecules, has emerged as a potential solution. 52 , 79–91 (2020). Cancer Res.

Small Molecule 126

Small Molecule Drugs Immune Response Targeted Protein Degradation 126

The Pharma Data

AUGUST 30, 2021

3,4 HFpEF has been described as the single largest unmet need in cardiovascular medicine based on prevalence, poor outcomes and the absence of clinically proven therapies to date. If approved, empagliflozin would become the first clinically proven therapy across the full heart failure spectrum. Completion: 2020.

Trials 52

Trials Therapies Hospitals Treatment 52

Drug Target Review

OCTOBER 1, 2024

At the beginning of my career, during my scholarship, I worked for two years at a research institute where I set up a research project focused on the study of metronomic oral therapies for patients with metastatic breast cancer (BC). That paper, of which I was in the authorship, remains a current point of reference for that therapy usage.

Doctors 97

Doctors Therapies Science Pharma Companies 97

The Pharma Data

JULY 21, 2021

A BTD is intended to expedite the development and review of medications to treat a serious medical condition and is granted when preliminary clinical evidence indicates the investigational therapy may demonstrate substantial improvement over existing therapies. Breakthrough Therapy. Food and Drug Administration. 3 Cancer.Net.

Therapies 52

Therapies FDA Treatment Vaccine 52

The Pharma Data

FEBRUARY 24, 2022

Bayer announced today the initiation of the CONFIDENCE study, a Phase II, three-arm study that will investigate simultaneous initial combination therapy with finerenone and the SGLT2 inhibitor empagliflozin, compared with finerenone alone and empagliflozin alone respectively in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D).

Clinical Development 52

Clinical Development Disease Therapies Treatment 52

LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline.

Small Molecule 140

Small Molecule Trials Therapies Disease 140

Creative Biolabs

JUNE 29, 2023

GPCR activity status detection In an article published online in 2020 by Bryan L. This provides new tools for diagnostic and therapeutic research in the field of neurology. Roth et al., based on BRET technology, single domain antibodies can be used to detect the activation state of receptors in cells in real time.

Therapies 52

Therapies Small Molecule Drugs Drug Development 52