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.

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Broad Institute

OCTOBER 11, 2023

Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. His mother had a presentation of the disease that suggested her immune system was already on the job.

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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.

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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.

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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.

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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.

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The Pharma Data

AUGUST 5, 2020

. Novartis has revealed positive results from a phase 2 study of its CAR-T therapy Kymriah (tisagenlecleucel) in patients with relapsed or refractory follicular lymphoma. The therapy is a one-time treatment created individually for each patient using their own T cells.

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The Pharma Data

JANUARY 11, 2021

Pfizer has started dosing in a phase 3 study evaluating its investigational gene therapy, PF-06939926, in young male patients with Duchenne muscular dystrophy (DMD). The agency previously granted the gene therapy Orphan Drug and Rare Pediatric Disease designations in 2017. Source link.

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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).

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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 team also observed that the drug prevented in vitro cancer stem cells from self-renewing, which is a characteristic of aggressive cancer cells that supports treatment resistance.

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Treatment Clinical Trials Science Research 105

The Pharma Data

DECEMBER 29, 2020

Primary Aldosteronism Testing Rare in Treatment-Resistant HTN. 29, 2020 — Patients with apparent treatment-resistant hypertension are rarely tested for primary aldosteronism, according to a study published online Dec. Veterans Health Administration with apparent treatment-resistant hypertension from 2000 to 2017.

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Treatment International Therapies Research 52

FDA Law Blog: Biosimilars

DECEMBER 4, 2023

A prescription not issued in the usual course of professional treatment or in legitimate, authorized research is not a prescription within the meaning of the CSA and the pharmacist knowingly filling it, along with the prescriber issuing it, is subject to penalties. 21 U.S.C. § 829; 21 C.F.R. Complaint ¶ 55. Complaint ¶ 58. Complaint ¶ 59.

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Drug Target Review

AUGUST 16, 2023

Cancer remains a major global health concern, often diagnosed at advanced stages when treatment options and survival rates are limited. Suspicious findings can prompt further evaluation and definitive diagnosis, enabling timely treatment and improved patient outcomes. References 1. Jillian Phallen. DOI:10.1126/scitranslmed.aan2415

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DNA Disease Therapies Treatment 98

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.

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Advarra

JUNE 12, 2023

Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. What Does an IBC Review? The post IBC vs. IRB: What’s the Difference? appeared first on Advarra.

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The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc. MUNICH, Germany, Jan.

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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.

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Therapies FDA Science Treatment 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.

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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. Sanofi and Sobi collaborate on the development and commercialization of efanesoctocog alfa.

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Drug Target Review

OCTOBER 17, 2023

To improve its treatment, early diagnosis and appropriate therapeutic strategies are very important. Diagnosis and therapy The study’s findings have promising implications for liver cancer therapy. 2017 July [cited 2023 October 9]; 3(2):45-51. Our immune cells depend on arginine to function properly,” Mossman explained.

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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

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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.

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Broad Institute

OCTOBER 4, 2023

The molecule’s dual mechanism of action — targeting both tumor and immune cells — is unique compared to other cancer immunotherapies including PD-1 drugs, and the researchers think it could explain why the molecule was so effective on its own in animal models and may not even need to be used in combination with other drugs such as anti-PD-1 therapy.

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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.

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Treatment Therapies Clinical Development Cell Biology 98

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 ®.

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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.

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Drug Target Review

OCTOBER 24, 2023

Live Bacterial Products (LBPs) will provide patients with a desirable, credible, safe and effective treatment option. The human microbiome, a complex ecosystem of microorganisms residing within our bodies, has emerged as a promising frontier in the quest for innovative medical treatments.

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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.

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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.

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Treatment Pharmacokinetics Therapies FDA 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).

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Treatment DNA Trials Therapies 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. Stem cell-derived organoids and their application for medical research and patient treatment.

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Clinical Research Research Drug Development Molecular Biology 116

The Pharma Data

JUNE 22, 2023

Kite Announces Completion of Marketing Authorization Transfer for Yescarta® CAR T-cell Therapy in Japan SANTA MONICA, Calif.–(BUSINESS in December 2022 about changes to their initial 2017 partnership whereby Daiichi Sankyo obtained the Marketing Authorization for Yescarta ®. –(BUSINESS WIRE)– Kite Pharma, Inc.,

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Common Sense for Drug Policy Blog

JANUARY 4, 2024

Cost Benefit Analysis of Opioid Treatment, Syringe Service Programs, and Test & Treat "Although model projections can only provide estimates of health benefits and costs, such analyses can provide intuition around critical mechanisms and assumptions to inform decision making. Published 2017 May 24. 2017;14(5):e1002312.

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The Pharma Data

JANUARY 17, 2021

The companies will evaluate ViGeneron’s proprietary, intravitreally injected vgAAV vectors for delivering a novel therapeutic protein to develop a gene therapy treatment for a highly prevalent eye disease. There is significant unmet medical need for a sustained therapy to treat eye diseases. MUNICH, Germany, Jan.

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New Drug Approvals

NOVEMBER 13, 2023

WO 2017/208267A1, WO 2020/250133 Al and WO 2022/024072A1, which are hereby incorporated by reference, disclose methods of use of the compound of Formula I for the treatment of Parkinson’s disease, synucleinopathies and Alzheimer’s disease (AD) respectively. amcrasto@amcrasto /////////// Ref [1].

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Treatment Disease Therapies Production 62

KIF1A

JUNE 17, 2023

This is why direct comparisons of different KIF1A mutations are invaluable; our patient-derived iPSC cells were developed for such comparisons, and projects like the Natural History and EEG studies help us find clinical patterns in KAND, so we can target therapies to the appropriate patients.

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The Pharma Data

JUNE 30, 2021

Event-free survival is defined as time from randomization to disease progression, commencement of new lymphoma therapy, or death from any cause. Safety results from the study were consistent with or lower than the known safety profile of Yescarta for the treatment of LBCL in the third-line setting. View the full release here: [link].

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