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

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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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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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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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

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

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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Alta Sciences

JUNE 27, 2023

Cancer Treatments, Clinical Trials, and the Motivation to Innovate nbartlett Tue, 06/27/2023 - 20:58 Getting to the Heart of Science with Michelle Newby At the heart of science are stories—stories of courage, challenges, successes, lessons, and people. Q: What was your cancer diagnosis and was treatment options were you given?

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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

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

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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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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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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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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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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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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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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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 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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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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The Pharma Data

JUNE 25, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for lecanemab (BAN2401), an investigational anti-amyloid beta (A?) protofibril antibody for the treatment of Alzheimer’s disease (AD). 1 The proof-of-concept Study 201 explored the impact of treatment with lecanemab on reducing brain amyloid beta (A?)

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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

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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The Pharma Data

OCTOBER 26, 2020

(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. In addition, the FDA has granted Fast Track designation for PR001 for the treatment of Parkinson’s disease with GBA1 mutations.

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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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The Pharma Data

JUNE 7, 2022

based production in support of its COVID-19 oral treatment, PAXLOVID TM (nirmatrelvir [PF-07321332] tablets and ritonavir tablets). increasing Pfizer’s capability to produce and supply treatments and medicines for patients in the U.S. and has manufactured almost 17 million treatment courses total. and around the world.

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The Pharma Data

OCTOBER 18, 2020

(Janssen) has announced it received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending the expanded use of Tremfya (R) (guselkumab) for the treatment of adult patients with active psoriatic arthritis (PsA) in the European Union (EU). The focus is on cancer.

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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.,

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PPD

OCTOBER 12, 2023

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. These treatments use a vector to introduce the desired nucleic acid code to replace or modify protein expression or use cells to alter/restore a specific cell type.

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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

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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The Pharma Data

MAY 18, 2021

Health Canada approves DUPIXENT® (dupilumab injection) as the first biologic for the treatment of moderate-to-severe atopic dermatitis in children aged 6 to 11 years. 9 DUPIXENT ® is also approved in Canada for the treatment of adult patients with severe CRSwNP and for the treatment of severe asthma in patients 12 years and older.

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New Drug Approvals

JANUARY 31, 2024

Thus, treatment of advanced prostate cancer involves androgen deprivation therapy (ADT) such as hormonal manipulation using gonadotropin-releasing hormone (GnRH) agonists/antagonists or surgical castration, AR antagonists or CYP17A1 inhibitors (such as abiraterone acetate in combination with prednisone). et al, Cancer Res.,

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Elrig

MARCH 12, 2024

Steve’s Keynote: Supporting the UK’s globally competitive innovation ecosystem There is a fundamental shift in how life science research is being carried out, the computational tools are transforming early drug discovery which in turn leads to real chemistry, advanced diagnostics and innovative therapies.

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Agency IQ

AUGUST 16, 2024

Many commenters took issue with the guidance’s statement that, “The contribution of the psychotherapy component to any efficacy observed with psychedelic treatment has not been characterized. Throughout 2017, Lykos sought feedback on the protocol for its first Phase 3 study, MAPP1, via the Special Protocol Assessment (SPA) process.

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The Pharma Data

JANUARY 20, 2021

20, 2021 — In what may signal a seismic shift in the treatment of methamphetamine addiction, a pair of prescription drugs appears to help patients significantly reduce their stimulant use, or quit altogether. Standard behavioral treatment for meth addiction typically involves highly structured group therapy, Volkow explained.

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Drug Target Review

APRIL 3, 2024

Our company has identified a new role for Growth Differentiation Factor 15 (GDF-15) as a potent local immunosuppressor mediating cancer resistance to therapy. Visugromab is a monoclonal antibody – a treatment modality that has been extensively studied and de-risked.

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