Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Could you give us an overview of the Lineage platform?

Therapies 84

Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

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. Lancet Haem 11: e693-e707. Shen et al.

Immune Response 67

Immune Response Therapies Treatment Disease 67

Drug Target Review

NOVEMBER 6, 2023

Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. 2 It is this complexity that necessitates powerful, targeted combination therapies.

Small Molecule 111

Small Molecule Therapies Treatment Disease 111

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

Eye on FDA

AUGUST 4, 2021

Each committee is comprised of a number of experts, diverse for their expertise, geography and specialty – with some focused on statistics while others may be focused on areas of concentration within the category, such as the treatment of pediatric patients. Fewer meetings – more approvals.

FDA 122

FDA Clinical Pharmacology FDA Approval Therapies 122

Drug Target Review

JANUARY 25, 2024

These findings offer a novel strategy for developing targeted treatments for triple-negative breast cancer, which currently has no approved options. The peptide in development is based on a 2018 Cleveland Clinic discovery and serves as a proof-of-concept for this type of drug for triple-negative breast cancer. Available from: [link]

Treatment 105

Treatment Clinical Trials Science Research 105

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

FDA Law Blog: Drug Discovery

JULY 17, 2024

An Opportunity to Leverage the Newly Created Rare Disease Advisory Committee across CDER and CBER In Frank and James’ 2018 proposal there was also a recommendation for the formation of a Rare Disease Advisory Committee, which would allow FDA access to experts in the science of small trials and other aspects of rare disease research.

FDA 105

FDA Disease Clinical Pharmacology Science 105

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

The Pharma Data

NOVEMBER 9, 2020

Talaris Chief Executive Officer Scott Requadt sees FCR001, the company’s investigational cell therapy, as a potential pipeline in a product (an experimental treatment that could have multiple uses across a number of indications). When that unit dissolved, rights to FCR001 reverted to Regenerex.

Therapies 52

Therapies Production Disease Treatment 52

The Pharma Data

DECEMBER 22, 2020

Tagrisso Approved as Adjuvant Therapy for NSCLC With EGFR Mutations. 22, 2020 — Tagrisso ( osimertinib ) received approval as an adjuvant treatment for patients with non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, the U.S. Professional. TUESDAY, Dec.

Therapies 52

Therapies Treatment FDA Disease 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

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

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. 2018): Ubiquitin Proteasome pathway proteins as potential drug targets in parasite Trypanosoma cruzi, Scientific Reports. (8):8399. link] Ojewale Kayode (2018).

Drugs 111

Drugs RNA DNA Disease 111

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 11, 2021

-based subsidiary of Terumo and a global neurovascular company, announced today the FDA Approval of the PMA Supplement for the WEB 17 System, a new addition to the WEB Aneurysm Embolization System for the treatment of intracranial wide neck bifurcation aneurysms. The WEB System received its first PMA approval in late 2018.

FDA Approval 52

FDA Approval Treatment FDA Therapies 52

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

The Pharma Data

OCTOBER 26, 2020

Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . EYSUVIS is the first FDA-approved corticosteroid specifically for dry eye disease treatment. Over-the-counter treatments fail due to the body’s natural protective system. 25% for dry eye disease treatment.

Treatment 52

Treatment Disease FDA FDA Approval 52

PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

FDA Approval 52

FDA Approval Therapies FDA Disease 52

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

JULY 8, 2021

AstraZeneca’s Biologics License Application (BLA) for tezepelumab has been accepted and granted Priority Review for the treatment of asthma from the US Food and Drug Administration (FDA). Tezepelumab received Breakthrough Therapy Designation for patients with severe asthma, without an eosinophilic phenotype in September 2018.

Treatment 52

Treatment FDA Clinical Trials Trials 52

The Pharma Data

DECEMBER 3, 2020

The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing eight weeks of treatment with either maribavir or investigator assigned treatment (IAT) in transplant recipients with CMV infection refractory or resistant to existing antiviral treatments (i.e.,

Treatment 52

Treatment Pharmaceutical Companies Therapies Virus 52

The Pharma Data

NOVEMBER 24, 2020

25, 2020 /CNW/ – On August 21, 2020 , Health Canada approved Lynparza® (olaparib), for the treatment of adult patients with deleterious or suspected deleterious germline and/or somatic BRCA or ATM- mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment with a new hormonal agent (NHA).

Treatment 52

Treatment DNA Trials Therapies 52

Broad Institute

JANUARY 22, 2024

Since 2018, the Cancer Dependency Map (DepMap) Consortium , an academic-industrial partnership launched by the Broad, has uncovered several potential drug targets by systematically screening cancer models in search of genetic dependencies. By Allessandra DiCorato January 22, 2024 Bill Sellers is the head of Broad's Cancer Program.

Drugs 128

Drugs Therapies Regulations RNA 128

The Pharma Data

JUNE 29, 2021

ENSPRYNG is the first and only NMOSD treatment that is administered subcutaneously every four weeks, allowing home-dosing after appropriate training. Importantly, people with NMOSD now have the flexibility to administer treatment at home, which may alleviate the need to travel for hospital appointments.”.

Treatment 52

Treatment Therapies Disease Clinical Development 52

The Pharma Data

SEPTEMBER 18, 2020

Tecentriq in combination with Avastin was also recently included as a class I, A recommendation by the European Society for Medical Oncology (ESMO) for the treatment of unresectable HCC, as well as by many clinical practice guidelines globally. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

Treatment 52

Treatment Therapies Disease Pharmaceuticals 52

The Pharma Data

MARCH 3, 2021

s (NYSE: PFE) supplemental New Drug Application (sNDA) for LORBRENA ® (lorlatinib), expanding the indication to include first-line treatment of people with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC). The FDA action also converts the 2018 accelerated approval to full approval. PRESCRIBING INFORMATION.

Treatment 52

Treatment FDA Approval FDA Disease 52

The Pharma Data

JANUARY 25, 2021

26, 2021 /PRNewswire/ — Dynacure , a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced today that the U.S. STRASBOURG, France and PHILADELPHIA , Jan. ” The U.S. ” The U.S.

Treatment 40

Treatment Disease Pharmaceuticals FDA 40

The Pharma Data

AUGUST 19, 2021

The Janssen Pharmaceutical Companies of Johnson & Johnson today announced preliminary data from the Phase 1 CHRYSALIS study evaluating RYBREVANT TM (amivantamab-vmjw) for the treatment of patients with non-small cell lung cancer (NSCLC) with mesenchymal-epithelial transition (MET) exon 14 skipping (METex14) mutations. 4] , [5] , [6].

Treatment 52

Treatment FDA Approval Pharmacokinetics FDA 52

The Pharma Data

MARCH 13, 2021

Takeda Pharmaceutical Company Limited ( TSE:4502/NYSE:TAK ) (“Takeda”) today announced that it has submitted a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for lanadelumab subcutaneous injection, a monoclonal antibody therapy for prophylaxis against attacks of hereditary angioedema (HAE).

Treatment 52

Treatment Pharmacokinetics Drugs Therapies 52

Drug Target Review

JANUARY 17, 2024

This insight could lead to potential therapies to prevent treatment resistance. Furthermore, the stem cell-like characteristics promote resistance to treatment through upregulation of efflux transporters, promotion of glioblastoma stem cell proliferation in neurogenic zones, and immune suppression.

Treatment 59

Treatment Therapies Disease Drugs 59

Drug Target Review

JULY 7, 2023

When targeted gene editing became a reality more than ten years ago, many people hoped for a revolutionary tsunami of new medical treatments. Epigenetic editing combines the power of multiplexing with the precise control of expression needed to expand the reach of gene therapies, enhance cell therapies, and enable genetic medicine.

DNA 105

DNA Engineering Therapies Regulations 105

The Pharma Data

SEPTEMBER 9, 2020

CERENOVUS Launches New Suite of Technologies to Advance Stroke Treatment. CERENOVUS Launches New Suite of Technologies to Advance Stroke Treatment. The announcement was made during the virtual European Society of Minimally Invasive Neurological Therapy (ESMINT). Stroke Research and Treatment, Hindawi, 27 Nov.

Treatment 52

Treatment Production Science Disease 52

Drug Target Review

JUNE 21, 2023

Enabling the systematic discovery of these largely uncharted targets can be a valuable opportunity for development of novel therapies. Genetic-based therapeutics hold significant promise in the clinic Despite the growing number of genes linked to human disease, the proportion of genes targeted by approved therapies remains low.

Disease 111

Disease Clinical Trials Therapies Science 111

The Pharma Data

JUNE 30, 2021

The EC’s decision is based on results from the Phase 2 CheckMate -142 trial in which Opdivo plus Yervoy demonstrated a clinically meaningful improvement in objective response rate (ORR) in patients with MSI-H/dMMR mCRC who received prior treatment with fluoropyridine, oxaliplatin and irinotecan. achieving a complete response.

Treatment 52

Treatment Trials FDA Approval Disease 52

Codon

JULY 14, 2024

The transfusion of platelets thus seems like an obvious treatment to stem bleeding. From Early Transfusion to Component Therapy The earliest documented attempts at blood transfusions involved the transfer of blood from animals to humans in the 17 th century. Component therapy offers several advantages over whole blood.

Production 141

Production Clinical Trials Trials Therapies 141

The Pharma Data

JUNE 28, 2021

Broadens company’s oncology platform of Targeted Alpha Therapies / Acquisition includes actinium-225 labeled differentiated PSMA small molecule for the treatment of prostate cancer. The pre-IND program focuses on the treatment of prostate cancer, the second most commonly diagnosed cancer in men. Noria) and PSMA Therapeutics Inc.

Small Molecule 52

Small Molecule Licensing Licensing Therapies 52