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. In some cases, cell therapy can be curative for the patient.

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Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

Eye on FDA

JANUARY 12, 2022

During nearly all of 2021, FDA has been under the direction of an acting commissioner. COVID – 144 announcements were COVID-related in 2021, compared to 274 such announcements during 2020; Approvals – there were 77 announcements related to approvals this year, 22 of which were COVID-related announcements.

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Drug Discovery Today

JANUARY 25, 2021

OSLO/LONDON, JANUARY 20 2021: EXACT THERAPEUTICS AS (“EXACT-Tx” or the “Company”), a clinical stage precision medicine company today announced a collaboration with GE Healthcare to develop an innovative ultrasound probe to be used in forthcoming studies using Acoustic Cluster Therapy (ACT®) across multiple disease conditions.

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Therapies Disease 113

Drug Discovery Today

MAY 11, 2021

Oslo, Norway, May 10, 2021 – Lytix Biopharma AS, a Norwegian clinical-stage immunoncology therapeutic company developing oncolytic molecule therapies to treat cancer, today announces that promising full data results from a phase I study of its lead candidate LTX-315 were published in Clinical Cancer Research, a peer-reviewed journal of the American (..)

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Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects.

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Small Molecule Drugs Clinical Pharmacology Trials 145

The Pharma Data

AUGUST 3, 2021

Revenue in the second quarter of 2021 increased 23 percent, driven by volume growth of 22 percent. – Revenue from all key products grew in the quarter and 2021 year-to-date. . – Revenue from all key products grew in the quarter and 2021 year-to-date. – 2021 EPS guidance updated to be in the range of $6.73

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Production Marketing Research DNA 52

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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Therapies Immune Response DNA Virus 69

Broad Institute

JUNE 10, 2024

The advance, from the lab of Broad core institute member David Liu , could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene. Tags: Gene editing Gene therapy David Liu Nature Biomedical Engineering.

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DNA Engineering Therapies Disease 144

KIF1A

APRIL 25, 2024

She is scheduled to be the 2nd patient in the world to receive ASO therapy for KAND. Timeline: December 2021 – We met with Dr Chung in NYC and discussed and applied to n-Lorem. March 2023 – We met with n-Lorem and found out Susannah’s ASO (1st KAND patient to receive ASO therapy for KAND) would work for Sloane.

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Therapies FDA Approval FDA Treatment 52

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Therapies Clinical Trials FDA Production 52

The Pharma Data

OCTOBER 14, 2021

The results, which demonstrate that patients treated with Biogen’s portfolio of MS therapies mount an effective antibody response to COVID-19 vaccination, are being presented at the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) virtual meeting, October 13-15, 2021.

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Vaccine Therapies Disease Immune Response 52

Drug Target Review

JULY 11, 2024

To date, there are less than 30 known surface proteins that form the basis of all approved cancer-targeted therapies of all modalities, including antibody-drug conjugates (ADCs), T-cell engagers, CAR T-cells and radiopharmaceuticals. These could be targets for antibody-drug conjugates (ADCs), bi-specific antibodies, or other therapies.

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Therapies RNA Pharma Companies Disease 56

Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% As more therapies are moving out of the lab and into human clinical trials, this paradigm is changing.

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Therapies Research Clinical Trials Trials 52

The Pharma Data

AUGUST 7, 2021

In June 2021, Wegovy , semaglutide 2.4 For the 2021 outlook, sales growth is now expected to be 10-13% at CER (previously 6-10%), and operating profit growth is now expected to be 9-12% at CER (previously 5-9%). ?? Lars Fruergaard Jørgensen, president and CEO: “We are pleased with the sales growth in the first half of 2021.

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International Treatment FDA Therapies 52

FDA Law Blog: Drug Discovery

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” It finalized a draft guidance published in 2022.

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Therapies FDA Production Regulations 59

The Pharma Data

SEPTEMBER 29, 2021

Presentations: 535P, 536P, 534P, 104P, 1534P, 444P, 630P, LBA26, 623P, 603P, 593P, 594P, 1309P, 606P, 590P, 826O, 530P, LBA58, 1520O, 48P, 508TiP, 505TiP, 509TiP, 1438TiP Bayer will present new data from its established portfolio of approved oncology therapies at the upcoming ESMO Congress 2021 from September 16-21, 2021.

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Research Clinical Trials Therapies Trials 52

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

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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Small Molecule Therapies Regulations Treatment 105

Eye on FDA

AUGUST 4, 2021

Below is a chart that looks at 3 numbers – the number of AdComms; the number of new molecular entities approved each year, and the number of drugs that had breakthrough therapy designation. That said, BT would be a lagging indicator since it can be granted so early in the development process. Fewer meetings – more approvals.

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FDA Clinical Pharmacology FDA Approval Therapies 122

The Pharma Data

SEPTEMBER 11, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. o Completion: 2021. o Number of patients: 3,730.

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Therapies Hospitals Clinical Trials Trials 52

Chemical Biology and Drug Design

OCTOBER 10, 2023

million people worldwide were living with HIV in 2021. This study focuses on NRTIs and NNRTIs, their binding sites, mechanisms of action, FDA-approved drugs and drugs in clinical trials, their resistance and adverse effects, their molecular docking studies, and highly active antiretroviral therapy (HAART).

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FDA Approval Clinical Trials FDA Virus 100

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. Biomaterials 273 , 120817 (2021).

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Small Molecule Drugs Immune Response Targeted Protein Degradation 126

The Pharma Data

SEPTEMBER 27, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with coronary failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. 2,3 a number one explanation for hospitalization within the U.S.

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Therapies Clinical Trials Hospitals Trials 52

The Pharma Data

AUGUST 5, 2021

The transaction was announced on July 19, 2021. The acquisition strengthens BioNTech’s cell therapy pipeline by accelerating the individualized solid tumor Neoantigen TCR cell therapy research and development program. Kite’s singular focus is cell therapy to treat and potentially cure cancer.

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Therapies Small Molecule Vaccine Science 52

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. In 2021, he assumed the role of Director in Protein Therapeutics at Gilead, driving biologics to prominence. Molecular Therapy 20 , 1831-1832 (2012). Nature 578 , 229-236 (2020).

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Engineering Virus Therapies Immune Response 105

The Pharma Data

SEPTEMBER 8, 2021

today announced that data reinforcing the potential of immunotherapies to impact clinical outcomes for patients will be featured at the European Society for Medical Oncology (ESMO) 2021 Virtual Congress from September 16-21. Summary of Presentations. All times noted are Central European Summer Time (CEST). Abstract Title. Presentation.

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Research Immune Response Treatment Trials 52

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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Research Immune Response Clinical Trials Therapies 137

The Pharma Data

MAY 26, 2023

State-of-the-art cryogenic freezers alongside material transfer equipment have been installed that are designed to retain the integrity of investigational advanced therapy products by minimizing their time-out-of-environment.

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Clinical Supply Therapies Clinical Trials Packaging 52

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. 2021 Jul 6;120(13):2644-56. 2021 Dec 1;9(12):1425-38.

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Treatment Therapies Clinical Development Cell Biology 98

Codon

APRIL 9, 2023

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. Read Ocular stress enhances contralateral transfer of lenadogene nolparvovec gene therapy through astrocyte networks. Molecular Therapy. Gene Therapy. McGrady N.R.

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The Pharma Data

MARCH 16, 2022

Dasha Nelidova (Institute of Molecular and Clinical Ophthalmology Basel) for developing a revolutionary therapeutic approach to restore light sensitivity in the eye retina of patients suffering from age-related macular degeneration by combining gene therapy and nanotechnolgy. billion euros. R&D expenses before special items amounted to 5.3

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Cell Biology Molecular Biology Science Laboratories 52

The Pharma Data

SEPTEMBER 28, 2021

Roche today announced data from across its growing neuromuscular portfolio at the planet Muscle Society (WMS) Virtual Congress 20 – 24 September 2021. We are grateful for the partnerships that are helping us to develop new therapies for people impacted by these devastating rare diseases.”. About SRP-9001 SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)

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Clinical Trials Protein Expression Therapies FDA 52

The Pharma Data

AUGUST 30, 2021

Results from the Phase III EMPEROR-Preserved trial were presented today at the European Society of Cardiology Congress 2021 2 and published in The New England Journal of Medicine 1. If approved, empagliflozin would become the first clinically proven therapy across the full heart failure spectrum. Completion: 2021.

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Trials Therapies Hospitals Treatment 52

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. Reference 1.

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The Pharma Data

MAY 27, 2022

1 Findings presented at the American Society of Clinical Oncology (ASCO) 2021 Annual Meeting showed that at a median duration of 18 months follow-up (range, 1.5–30.5), of patients with RRMM responding to therapy (98 percent overall response rate ORR ).1,6 In December 2017, Janssen Biotech, Inc. to develop and commercialise cilta-cel.[1].

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Therapies Treatment Pharmaceutical Companies Nurses 52

The Pharma Data

SEPTEMBER 30, 2021

As one among the leading global vaccines companies, we still bring options which help protect people’s health round the world,” said Michael Greenberg, MD, MPH, Medical Head, Sanofi Pasteur North America. Nirsevimab has been granted breakthrough designation by four major regulatory agencies round the world.

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Vaccine Research Virus Disease 52

The Pharma Data

SEPTEMBER 11, 2021

– Designation is based on results from the EMPEROR-Preserved phase III trial, which established Jardiance as the first therapy to show statistically significant improvement in heart failure outcomes in adults with heart failure with preserved ejection fraction. vice president, Product Development, Lilly. Median follow-up time was 26.2

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