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. Molecular Therapy-Nucleic Acids. Human molecular genetics. 2014 Jan 1;3.

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SCIENMAG: Medicine & Health

AUGUST 26, 2023

In 2017, the teams of Massimo Terzolo and Martin Fassnacht published a study in the New England Journal of Medicine that provided evidence for the efficacy of Mitotane in the prevention of recurrence in adrenocortical carcinoma.

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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Broad Institute

OCTOBER 11, 2023

But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way. His mother had a presentation of the disease that suggested her immune system was already on the job.

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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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Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

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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. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Molecular Therapy 20 , 1831-1832 (2012). The Lancet 390 , 849-860 (2017).

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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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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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Drug Target Review

MARCH 15, 2024

2017 Nov;33(6):1468-1475. Epub 2017 Sep 7. Cell Gene Therapy Insights 2017; 3(2), 131-158, 10.18609/cgti.2017.014. Leveraging a CHO cell line toolkit to accelerate biotherapeutics into the clinic. Biotechnol Prog. doi: 10.1002/btpr.2548. PMID: 28842948. Narayanavari SA, Izsvák Z. Patel T, Ingham C, Fleming J, et al.

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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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Therapies Virus Disease Licensing 52

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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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 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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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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Drug Target Review

JANUARY 15, 2024

Immunotherapy has become a cornerstone of cancer therapy and significantly improved patient outcomes Immunotherapy has become a cornerstone of cancer therapy and significantly improved patient outcomes The tumour regression at the metastatic site (abscopal effect) in the 4T1 tumour model is driven by CD8+ killer T cells.

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Drug Target Review

JANUARY 25, 2024

Triple-negative (ER-negative, PR-negative, HER2/neu not overexpressed) breast cancer has distinct clinical and pathologic features, and is a clinical problem owing to its aggressive behaviour and lack of targeted therapies that leave chemotherapy as the mainstay of treatment, resulting in a relatively poor prognosis.

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Drug Target Review

AUGUST 16, 2023

This in-depth understanding of the disease could aid in selecting targeted therapies for individual patients, further enhancing treatment outcomes. Future directions and prospects Moving forward, the researchers plan to analyse the molecular vibrational spectrum associated with various clinical characteristics of cancer. References 1.

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The Pharma Data

DECEMBER 21, 2020

Legend will receive $75 million from J&J as a clinical development-based milestone payment, per a 2017 collaboration pact. Cilta-cel also won a Breakthrough Therapy designation in China in August. Source link.

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Drug Target Review

OCTOBER 17, 2023

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. Therefore, depleting arginine in the tumour environment helps the tumour cells escape the immune system.”

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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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Drug Target Review

DECEMBER 16, 2024

Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.

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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. From 2017 to March 13, 2022, Biogen and Eisai jointly collaborated on the development, commercialization and manufacturing of ADUHELM.

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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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Drug Target Review

FEBRUARY 2, 2024

Compared to conventional models, could you explain how Organovo’s 3D tissue models facilitate a deeper understanding of genetic expression within specific diseases, and how this insight might transform the development of targeted therapies? Once created, our 3D human tissue models can be manipulated and worked with in the lab.

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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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The Pharma Data

MAY 27, 2022

In December 2017, Janssen Biotech, Inc. 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.,

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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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Laboratories Therapies Disease Research 98

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. 1 , 33–43 (2018). 102 , 1–6 (2016).

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Eye on FDA

FEBRUARY 8, 2024

The last time the agency issued less than 200 releases to the public was back in 2017. The number of releases in relation to new gene therapies increased from 3 to 5, however it is worth noting that one of the 2023 releases actually addressed the approval of 2 new gene therapies in one release.

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Creative Biolabs

JUNE 29, 2023

In 2017, a research team screened a series of single domain antibodies, which can recognize different conformations of receptors and also has agonist effects and positive allosteric effects.

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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. ZUMA-7 evaluated an one-time infusion of the CAR T-cell therapy Yescarta compared to SOC in adult patients with relapsed or refractory LBCL. View the full release here: [link].

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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. Human pluripotent-stem-cell-derived organoids for drug discovery and evaluation. Elsevier; 2020.

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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]. amcrasto@amcrasto /////////// Ref [1].

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Drug Target Review

AUGUST 24, 2023

Neutralising GDF-15 with CatalYm’s anti-GDF-15 antibody visugromab was shown to reverse its inhibitory effects and to re-sensitise tumours to anti-PD-1 treatment, achieving commensurate survival benefit of anti-GDF-15-anti-PD-1 combination therapy in vivo. in North America) in late 2017.

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Drug Target Review

SEPTEMBER 26, 2023

Early detection of neurotoxicity induced by potential new therapies is a major challenge, and hiPSC-neuronal cells may provide a solution. 2017 Jan;155(1):234–47. 2017 Jul 6;21(1):14–7. 2017 Feb;16(2):115–30. Toxicol Sci. Gintant G, Fermini B, Stockbridge N, Strauss D. Cell Stem Cell. Nat Rev Neurol. Nat Rev Drug Discov.

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FDA Law Blog: Biosimilars

DECEMBER 4, 2023

Prescribing Red Flags The government alleged that from at least 2017 to April 2021 Defendants knowingly filled controlled substance prescriptions “that raised obvious ’red flags’ of potential abuse or diversion.” If the pharmacist can resolve it, they must make a record of the resolution. Complaint ¶ 55. Complaint ¶ 58.

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