Codon

NOVEMBER 3, 2024

In the 1924 novel, The Magic Mountain , Thomas Mann describes a sanatorium patient named Anton Ferge as he undergoes a painful tuberculosis (TB) treatment. These challenges have led to strategies such as directly observed therapy (DOT), in which nurses or physicians monitor patients to ensure they take their medicine every day.

Vaccine 102

Vaccine Trials Therapies Treatment 102

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options.

Small Molecule 145

Small Molecule Drugs Clinical Pharmacology Trials 145

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 67

Immune Response Therapies Treatment Disease 67

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

Eye on FDA

MARCH 5, 2023

Note these numbers cover meetings about drug applications only, not including vaccines or cellular therapies. That said, beginning in 2020 meetings began skewing to more negative outcomes and 2022 was the first year where the number of negative outcomes outweighed the number of positive outcomes.

FDA 72

FDA Vaccine Drugs Therapies 72

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. It may also limit coverage for any future approved treatment in the class. ADUHELM is indicated for the treatment of Alzheimer’s disease.

Treatment 52

Treatment Therapies Disease FDA Approval 52

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. This may lead to a more robust restoration of muscle function.

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

The Pharma Data

MAY 27, 2022

In the pivotal clinical study CARTITUDE-1, 98 percent of patients with relapsed or refractory multiple myeloma responded to a one-time treatment with ciltacabtagene autoleucel and 80 percent of patients who responded experienced a stringent complete response . In December 2017, Janssen Biotech, Inc. to develop and commercialise cilta-cel.[1].

Therapies 52

Therapies Treatment Pharmaceutical Companies Nurses 52

National Institute on Drug Abuse: Nora's Blog

MARCH 6, 2025

Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&

Treatment 69

Treatment Trials Clinical Trials Drugs 69

Drug Target Review

NOVEMBER 7, 2023

Chemists at Stanford University discovered how one of the pathways leading to this protein “woodchipper” works furthering the understanding of new therapeutics for autoimmune diseases, age-related disorders, and treatment-resistant cancers. Enzyme replacement therapy is a frequent treatment for these disorders.

Therapies 59

Therapies Science Disease Treatment 59

DrugBank

AUGUST 9, 2024

However, searching for broader applicability and personalized approaches continues, as not all patients respond to these treatments. These new therapies have demonstrated significant LDL cholesterol reductions, but their high cost remains a barrier to widespread adoption.

Pharmaceuticals 98

Pharmaceuticals Research Pharmaceutical Companies Clinical Trials 98

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

The Pharma Data

MARCH 9, 2022

Investigators at Cedars-Sinai have identified a potential new therapy for COVID-19: a biologic substance created by reengineered human skin cells. Although still in the early stages, the findings open the possibility of having a new therapy for COVID-19 patients.

Treatment 52

Treatment Virus Engineering Therapies 52

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

The Pharma Data

MARCH 24, 2021

billion EUR in 2020 (18.9% Boehringer Ingelheim stepped up its investments in R&D significantly in 2020 in pursuit of innovative medicines and therapies for diseases for which no satisfactory treatments are available. In particular, efforts to research potential COVID-19 related therapies were accelerated.

Therapies 52

Therapies Disease Vaccine Small Molecule 52

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. Nature 578 , 229-236 (2020). References Doudna JA.

Engineering 105

Engineering Virus Therapies Immune Response 105

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 Trials 52

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

The Pharma Data

APRIL 17, 2021

TECFIDERA was approved under the National Medical Products Administration priority review process evaluating therapies with urgent clinical needs. Biogen’s expansion in China now includes treatment options approved for relapsing MS and spinal muscular atrophy. We thank the NMPA for undertaking priority review to approve TECFIDERA. “We

Treatment 52

Treatment Clinical Trials Disease Trials 52

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. 95: 729–38

Clinical Research 116

Clinical Research Research Drug Development Molecular Biology 116

The Pharma Data

JUNE 28, 2021

Parkinson’s disease is most common neurodegenerative movement disorder / It impacts more than 10 million people worldwide/ No function-restoring therapy is currently available / Bayer is pursuing a two-pronged approach to deliver transformative therapies with one cell and one gene therapy candidate in clinical trials.

Therapies 52

Therapies Disease Clinical Trials Trials 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

APRIL 15, 2022

Designed computationally and refined in the laboratory, the new protein therapies thwarted infection by interfering with the virus’ ability to enter cells. The top protein neutralized the virus with similar or greater potency than antibody treatments with Emergency Use Authorization status from the U.S. 2 omicron subvariant.

Treatment 52

Treatment Virus Laboratories Engineering 52

Drug Target Review

JULY 1, 2024

As a non-opioid, SRP-001 also eliminates abuse potential, positioning it as a safer and effective drug candidate for the treatment of acute and neuropathic pain and migraine headache. Could you elaborate on the potential market size for SRP-001 and how it fits into the broader landscape of pain therapies? South Rampart Pharma, Inc.

Treatment 59

Treatment Clinical Trials Therapies Molecular Biology 59

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

The Pharma Data

MARCH 30, 2021

Approval based on two Phase III ASCLEPIOS studies that met primary endpoints where Kesimpta showed a reduction of annual relapses by over 50% versus teriflunomide, a first-line treatment in MS, and achieved more than 30% relative risk reduction of 3-month confirmed disability progression 2.

Therapies 52

Therapies Treatment Disease Licensing 52

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

JUNE 28, 2021

Food and Drug Administration (FDA) and a marketing authorization application (MAA) to the European Medicines Agency (EMA) seeking approval of the novel combination of the cancer treatments copanlisib and rituximab. submission is for the treatment of patients with relapsed indolent B-cell non-Hodgkin’s lymphoma (B-iNHL). Fields, M.D.,

Treatment 52

Treatment Therapies Trials Clinical Development 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

AUGUST 15, 2021

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

Disease 52

Disease FDA Treatment Clinical Trials 52

The Pharma Data

MAY 4, 2021

Tecentriq monotherapy has been shown to improve overall survival in people with high PD-L1 expression, when compared to chemotherapy, and therefore represents a new treatment option for people living with this difficult-to-treat disease.”. Grade 3–4 treatment-related adverse events were reported in 12.9% months (HR=0.76, 95% CI: 0.54–1.09).

Treatment 52

Treatment Therapies Disease Pharmaceuticals 52

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. About the EMPEROR coronary failure studies10,11.

Therapies 52

Therapies Clinical Trials Hospitals Trials 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

MARCH 26, 2021

If approved, Tecentriq could offer people with a specific type of lung cancer a chemotherapy-free option in the first-line treatment setting. We remain committed to providing effective and tailored lung cancer treatment options, and this announcement is an important step toward this goal.”. months; hazard ratio [HR]=0.59, 95% CI: 0.40–0.89;

Treatment 52

Treatment Therapies Protein Expression Immune Response 52

The Pharma Data

APRIL 8, 2021

Since its first approval in renal cell carcinoma nearly two years ago, KEYTRUDA has become an important first-line treatment option in combination with axitinib for patients with advanced renal cell carcinoma,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories.

Therapies 52

Therapies Disease Research Laboratories Treatment 52

The Pharma Data

FEBRUARY 22, 2022

Food and Drug Administration (FDA) for the adjunctive treatment of major depressive disorder (MDD) in patients who are receiving ongoing antidepressant therapy. In 2020, an estimated 21 million adults had at least one major depressive episode. VRAYLAR is also approved for the treatment of schizophrenia in adults (1.5

Treatment 52

Treatment FDA Clinical Trials FDA Approval 52