Fierce BioTech

JULY 25, 2023

How combining datasets from multiple sources can inform and improve commercial strategies Hundreds of rare disease treatments have entered the market over the past decade—thanks largely to a combination of government incentives, strong urging from patient advocacy groups and advances in cell and gene therapies.

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Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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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 Premier Consulting Blog

APRIL 18, 2024

Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. Does it have to be in a disease model? The cell or gene therapy should be pharmacologically active in the animal model. The cell or gene therapy should be pharmacologically active in the animal model.

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SCIENMAG: Medicine & Health

JUNE 16, 2023

Breast cancer is a complex disease, and its progression is difficult, yet important, to predict. Credit: UMass Amherst Breast cancer is a complex disease, and its progression is difficult, yet important, to predict.

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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. Hearing aids also have all sorts of deficits.

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Broad Institute

JANUARY 25, 2024

They found significant associations between the thinning of different retinal layers and increased risk of developing ocular, cardiac, pulmonary, metabolic, and neuropsychiatric diseases and identified genes that are associated with retinal layer thickness. Their findings are published in Science Translational Medicine. “We

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DrugBank

JUNE 13, 2024

Similarly, for cardiovascular diseases, implantable drug-eluting stents can deliver controlled doses of anti-proliferative drugs directly to the site of arterial blockage, preventing restenosis (re-narrowing) and improving long-term integrity of the vessel. 

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The Premier Consulting Blog

APRIL 18, 2024

Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.

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SCIENMAG: Medicine & Health

AUGUST 7, 2023

Philadelphia, August 8, 2023 – There is a need to better deliver information on medical nutrition therapy for patients with Parkinson’s disease (PD).

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Agency IQ

JUNE 26, 2023

FDA quietly kicks off process that could inform future rare disease regulatory reforms Reforms buried within the FY2023 omnibus spending bill require the development of a new report looking at the regulatory processes of the U.S. Meanwhile, in the EU rare diseases are defined using a different set of criteria. In the U.S.,

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Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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Drug Target Review

NOVEMBER 23, 2023

This means the neuropeptide network probably uses different strategies to transmit and process information than a more hierarchical wired network does. elegans be applied to the development of targeted therapies for neuropsychiatric conditions like eating disorders, OCD, and PTSD? elegans ?

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thought leadership

JUNE 10, 2024

Cell and Gene Therapies (CAGTs) represent a transformative step in medicine, offering unprecedented potential to treat and potentially cure diseases once deemed untreatable. However, the path to accessing these advanced treatments can be daunting.

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FDA Law Blog: Drug Discovery

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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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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The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America. SATURDAY, Dec.

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The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

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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. ADUHELM is indicated for the treatment of Alzheimer’s disease. It may also limit coverage for any future approved treatment in the class.

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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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Drug Target Review

APRIL 4, 2024

What are the key findings of the preclinical study regarding the neurophysiological brain state in a Parkinson’s disease psychosis (PDP) model? The animal model is based on creating a dopamine-deficient state, by a specific lesion of dopaminergic cells, and then exposing it to repeated substitution therapy with L-DOPA.

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Drug Target Review

OCTOBER 10, 2024

What are the risks of over-reliance on healthy donor material in early-stage research, and how can decision-makers ensure a balanced integration of diseased material to avoid potential gaps when transitioning to clinical trials? Diseased cells are likely to be more fragile or less abundant, making it difficult to obtain enough for therapy.

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Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

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The Pharma Data

OCTOBER 9, 2021

Collaboration Leveraging Neumora’s Precision Neuroscience Platform with Amgen’s deCODE Human Genetics Capabilities to Advance Innovative Medicines for Brain Disease. Reese, M.D., executive vice president of Research and Development at Amgen.

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Agency IQ

FEBRUARY 12, 2024

BY RACHEL COE, MSC | FEB 7, 2024 10:21 PM CST The Bespoke Gene Therapy Consortium A collaborative effort, the Bespoke Gene Therapy Consortium (BGTC) was launched in October 2021 to “accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease.”

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Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

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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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DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

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The Pharma Data

JULY 8, 2021

ADUHELM should be initiated in patients with mild cognitive impairment due to Alzheimer’s disease or mild Alzheimer’s dementia. The update includes an addition to the Indications and Usage section of the label (Section 1) to emphasize the disease stages studied in the clinical trials, as seen below ( italics to note updated language).

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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.

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The Pharma Data

OCTOBER 26, 2020

DALLAS–( BUSINESS WIRE )– Taysha Gene Therapies Inc. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Obtaining these key designations highlights our commitment to developing a gene therapy for the treatment of SURF1 deficiency.”.

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The Pharma Data

MAY 31, 2022

the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). of BRT-DA01 in patients with Parkinson’s disease.?. More information about the Phase 1 trial is available at clinicaltrials.gov (NCT#04802733). The purpose of the?Ph1

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The Pharma Data

MAY 31, 2022

BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.

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Drug Target Review

OCTOBER 17, 2024

It is not known whether CD24 on cancer cells has a unique epitope that can be specifically targeted for cancer therapy. To explore its therapeutic potential in cancer and other immunological diseases, we developed an antagonist anti-Siglec-10 monoclonal antibody called ONC-841. Combination therapy is the best approach in immunotherapy.

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The Pharma Data

JANUARY 18, 2021

“There are no approved therapies for epilepsy caused by SLC13A5 that address the underlying cause of this disease,” said RA Session II, President, Founder and CEO of Taysha. “We Taysha’s commitment to developing a potentially life-changing gene therapy for SLC13A5 deficiency is greatly welcomed by our patient community.”.

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The Pharma Data

AUGUST 5, 2021

The acquisition strengthens BioNTech’s cell therapy pipeline by accelerating the individualized solid tumor Neoantigen TCR cell therapy research and development program. It also expands the Company’s cell therapy capabilities and manufacturing footprint in North America, building on its acquisition of Neon Therapeutics in 2020.

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Drug Hunter

MAY 21, 2023

These findings highlight the versatility of DCAF1 for TPD and offer a new avenue for developing therapies for diseases where catalytic inhibition is not an option. Drug Hunter Premium is drug discovery, distilled, so you can quickly catch up and make informed decisions based on industry examples. already a member?

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