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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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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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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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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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

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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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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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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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

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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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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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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Drug Patent Watch

JANUARY 9, 2025

Enter the Contract Development and Manufacturing Organization (CDMO) a lifeline for many startups looking to bring their innovative therapies to market. In 2017, small and medium-sized biotech companies accounted for 51% of FDA market approvals, while large pharma companies were the originators in only 28% of approvals[4].

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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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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

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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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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The Pharma Data

JANUARY 18, 2021

This technology has received a lot of attention in our facility, and, likely, many of the patients who need and then receive CCM therapy will ultimately require MRI procedures,” said Dr. Robert W. CCM therapy is the first approach designed to optimize heart contraction, allowing more oxygen-rich blood to reach the body.

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The Pharma Data

SEPTEMBER 10, 2020

Today’s approval is an important advance for these patients as it allows them to benefit from triple therapy by using one inhaler, once a day.”. Trelegy is a combination of three molecules in a single inhaler that needs to be taken once per day.

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The Pharma Data

NOVEMBER 15, 2020

As its name aptly suggests, Urovant is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for urologic conditions such as overactive bladder (OAB) and maladies involving adjacent areas of the human anatomy. .

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New Drug Approvals

SEPTEMBER 13, 2024

19] As of 2016, aticaprant has reached phase II clinical trials as an augmentation to antidepressant therapy for treatment-resistant depression. [20] 21] In August 2017, it was announced that Cerecor had sold its rights to aticaprant to Janssen Pharmaceuticals. [22] Dhir A (January 2017). August 2017. nM vs. 24.0

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Drug Target Review

MAY 23, 2023

Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. However, many patients respond poorly to corticosteroid treatment, or their immune system responds unfavourably to biological therapies, such as the development of autoimmune diseases.

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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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The Pharma Data

DECEMBER 29, 2020

Veterans Health Administration with apparent treatment-resistant hypertension from 2000 to 2017. There was an association noted between testing and an increased likelihood of initiating mineralocorticoid receptor antagonist (MRA) therapy (hazard ratio, 4.10) and with better blood pressure control over time. Cohen, M.D.,

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PPD

OCTOBER 12, 2023

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. Since chimeric antigen receptor T cell (CAR-T) therapy was first approved in 2017, there has been a marked increase of cell and gene therapy studies resulting in significant changes in the way diseases are treated as well as patient outcomes.

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The Pharma Data

MARCH 30, 2021

(NASDAQ:AMGN) and Rodeo Therapeutics Corporation (Rodeo) today announced an agreement under which Amgen will acquire Rodeo, a privately held biopharmaceutical company based in Seattle that develops small-molecule therapies designed to promote regeneration and repair of multiple tissues. senior vice president of Global Research at Amgen.

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Drug Target Review

MAY 18, 2023

He expects that most participants will be drawn from the pool of approximately 120 patients currently being treated at the Center for Blood Disorders, many of whom have declined hydroxyurea therapy. Kutlar says the possibilities of this additional therapy are exciting. Additionally, not all patients respond to every drug.

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The Pharma Data

AUGUST 9, 2020

Etrolizumab failed to meet its primary endpoint versus placebo as maintenance therapy in people with ulcerative colitis. References [1] GBD 2017 Inflammatory Bowel Disease Collaborators. 2017; 389(10080):1756-70. [3] 2017; 389(10080):1756-70. [3] 2017; 389:1741-55. [4] 2020; 5(1):17-30. [2] 2] Ungaro R, et al.

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The Pharma Data

MARCH 1, 2022

“We are excited to regain exclusive, worldwide rights to ASP3772, our MAPS vaccine candidate for Streptococcus pneumoniae that has achieved positive Phase 2 clinical trial results and received FDA Breakthrough Therapy designation. We believe ASP3772 has significant potential to address unmet needs in this important vaccine category.

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Common Sense for Drug Policy Blog

JUNE 6, 2024

Analogous substances like post-cycle therapy drugs and human growth hormone, on the other hand, are generally produced in licit pharmaceutical laboratories in jurisdictions with more lax regulations and subsequently imported or removed from domestic pharmaceutical supply chains by illicit actors (Fink et al. 2017 ; Greenway and Price 2018 ).

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Drug Target Review

OCTOBER 24, 2023

Could you elaborate on the drug being developed by Microbiotica to stimulate cancer patients to respond favourably to immune checkpoint inhibitor therapy? It is likely the combination of these mechanisms of action that will make MB310 an effective therapeutic. How does it work and what are the potential benefits? difficile and C.

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Alta Sciences

APRIL 17, 2024

Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.

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