The Pharma Data

AUGUST 4, 2020

Novartis is celebrating the success of its one-time CAR T therapy Kymriah (tisagenlecleucel) in patients with relapsed or refractory (r/r) follicular lymphoma (FL), revealing that the therapy met its primary its primary endpoint of complete response rate (CRR) in Phase 2 trials, according to an independent review committee.

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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

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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Conversations in Drug Development Trends

NOVEMBER 5, 2024

NC: I think the continued growth in cell and gene therapy research and the approvals we’re seeing for genetically-driven rare diseases. Both approaches need experts in the drug process, and CROs can definitely make an impact. Looking ahead to 2025, what are your hopes for rare disease research?

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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 It finalized a draft guidance published in 2022.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

MARCH 3, 2025

In this interview, Professor Schwamborn discusses his research, the promise of brain organoids, and his vision for the future of stem cell-based therapies. “We believe this will definitely make its way into preclinical research and practice.” Developments are moving quickly, and we’re making significant progress.

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FDA Law Blog: Drug Discovery

MAY 19, 2024

aimed at identifying and characterizing the challenges in developing therapies for ultra-rare diseases and conditions that affect exceedingly small populations. By Sarah Wicks — On May 21, 2024, the EveryLife Foundation for Rare Diseases (ELF) will host a Scientific Workshop at the National Press Club in Washington, D.C.

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The Pharma Data

JUNE 9, 2025

These results further validate the potential of Ascendis’ proprietary TransCon technology to deliver long-acting therapies that improve both efficacy and patient experience. Importantly, the combination therapy maintains a safety profile that aligns with what we’ve observed in monotherapy studies.” years in prior clinical studies.

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PPD

MARCH 11, 2025

While there is no universally accepted definition for rare disease, it is estimated that more than 10,000 distinct rare conditions exist, and people living with these rare diseases represent as much as 10% of the global population.

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Drug Target Review

APRIL 14, 2025

By definition, preventative drugs are taken by a larger number of customers because you never know exactly whos going to get an infection its probabilistic. Finrow notes that their intellectual property strategy required new types of patents and trade secrets, as spirulina-based therapies had not been fully explored in the biotech space.

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Drug Target Review

OCTOBER 30, 2024

In light of the natural composition of organs, a formal definition of an ‘organoid’ might be a mini organ in a dish that comprises all or most of the various cell types found in the organ being modelled. 22-26 Technically, these findings could apply to both restoration and enhancement of organ function.

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Drug Target Review

AUGUST 16, 2023

Suspicious findings can prompt further evaluation and definitive diagnosis, enabling timely treatment and improved patient outcomes. This in-depth understanding of the disease could aid in selecting targeted therapies for individual patients, further enhancing treatment outcomes. References 1. Jillian Phallen.

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FDA Law Blog: Biosimilars

APRIL 10, 2023

Houck — Drugs and substances classified within schedule I of the federal Controlled Substances Act (“CSA”) by definition have a high potential for abuse, no currently accepted medical use in treatment in the United States, and lack accepted safety for use under medical supervision. By Larry K. 21 U.S.C. § Mace & Dean with Sen.

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Drug Target Review

JANUARY 12, 2024

This one size fits all approach to drug prescribing still pervades in mostly all therapy areas except oncology. Therefore, having biomarker information is useful for separating individuals and ensuring that you treat an individual as an individual and can tailor their therapy. Most drugs do not work in all people.

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LifeSciVC

JULY 8, 2025

This essay takes up those questions, not to provide definitive answers, but to explore the contours of a new leadership landscape. Not because they’re definitive, but because they feel aligned with the realities biotech leaders now face. One team might be working on a cell therapy, another on RNA, another on small molecules.

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LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. ALT) continue to build momentum in the indication. Will incretins “solve” MASH in obese patients?

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LifeSciVC

APRIL 17, 2025

However, there are times when refusing to stop and re-evaluate is to our disadvantage, especially if our (and our teams) bandwidth can be better spent pursuing other potential therapies. Similarly, its encouraged to map out your key 1-3 programs before even running any experiments to validate your platform.

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PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.

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The Pharma Data

DECEMBER 29, 2020

a biotechnology company developing cell therapies that enable organ regeneration, announced today that the U.S Under the IND, LyGenesis will be conducting a Phase 2a study on the safety, tolerability, and efficacy of its first-in-class novel cell therapy for patients with end stage liver disease (ESLD). and Longevity Vision Fund.

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Drug Target Review

DECEMBER 4, 2023

It involves, almost by definition, the consolidation of extensive patient data: genomic information, health records and other external resources. 4 The potential impact on patient outcomes that this will enable is colossal, but further advancement must be made to make such therapies truly accessible.

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The Pharma Data

AUGUST 17, 2020

DF6002 has the potential to stimulate effective anti-tumor immunity in patients who are not eligible or not adequately responding to current therapies. DF6002, Dragonfly’s extended half-life IL12 cytokine, is an investigational immunotherapy being evaluated in adult patients for the treatment of advanced solid tumors. About Dragonfly.

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Broad Institute

OCTOBER 23, 2024

The work highlights the importance of examining the often-ignored noncoding parts of the genome when diagnosing rare genetic disorders, and also points to possible future genetic therapies that fine-tune CHASERR abundance as a means to treat CHASERR - and CHD2 -related disorders.

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KIF1A

JUNE 21, 2025

n-Lorem and ASO Therapies : Laury Mignon from n-Lorem will discuss their approach to ASO therapies and “n-of-1” treatment development. You’ll also hear directly from families currently receiving or preparing to receive ASO therapy. She’s definitely the Queen Bee! London was diagnosed with KIF1A in 2022.

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The Pharma Data

SEPTEMBER 8, 2021

Adds Rezurock™ (belumosudil) an FDA-approved, first-in-class treatment for adult and pediatric patients 12 years and older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy. Sanofi has entered into a definitive merger agreement with Kadmon Holdings, Inc. Transaction Terms.

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The Pharma Data

SEPTEMBER 23, 2020

The halting of AstraZeneca’s COVID-19 vaccine trial around the world was cause for much initial concern over the eventual safety and efficacy of the therapy. Trials have resumed in the UK, Brazil and South Africa, but US trials remain on hold. Matt Fellows. Source link.

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The Pharma Data

DECEMBER 22, 2020

(NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced that it will convene its 2020 Annual Meeting of Stockholders on December 23, 2020 at 11:00 a.m. The Annual Meeting will resume with respect to Item 2 at 11:00 a.m.

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Perficient: Drug Development

AUGUST 9, 2024

Understanding Touch and Gesture Accessibility Definition and Scope: Touch Accessibility: Ensures that touchscreen interfaces are designed to be easily operable by all users, including those with limited dexterity or motor control.

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The Pharma Data

OCTOBER 19, 2020

By combining the EMV+ ventilator with the SAROS 4000 Oxygen Concentrator, remote medics will be able to deliver high-end, oxygen-enriched critical care ventilation and oxygenation therapy without the use of oxygen cylinders, which present refilling and logistical challenges and are a hazardous threat in armed conflicts.

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Drug Target Review

OCTOBER 31, 2023

By definition, this is the cutting edge, and we are in it. We will be wrapping up an ongoing study and are hopeful that we can bring a therapy to market for this disease. Right now, I am focused on making sure this potential therapy is positioned to get the appropriate labels for commercialisation.

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The Pharma Data

MARCH 30, 2021

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

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The Premier Consulting Blog

NOVEMBER 8, 2022

Working to bring these valuable new therapies to patients, sponsors engaged in strategic development often approach Premier Consulting for regulatory clarification: “Is this actually a combination product? metered-dose inhaler) However, the four-part definition in 21 CFR 3.2(e) What do we need to do if it is?”

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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. By definition, the do-not-eat-me signal refers to phagocytosis of tumour cells by macrophages, although we and others have shown that the CD24/Siglec-10 pathway also regulates the function of T cells and NK cells.

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The Pharma Data

NOVEMBER 19, 2020

Neurotrope is a clinical-stage biopharmaceutical company working to develop novel therapies for neurodegenerative diseases. Neurotrope has entered into a definitive merger agreement pursuant to which Metuchen Pharmaceuticals, L.L.C. About Neurotrope, Inc.

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The Pharma Data

OCTOBER 27, 2021

Kymriah was preliminarily granted orphan medicinal product designation by the European Commission (EC) forFL.However, Kymriah would have the occasion to present an important treatment option for those cases with r/ r FL in need of potentially definitive issues, If approved in this implicit third suggestion.

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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. Breakthrough therapy designation: Gives priority review to therapies offering substantial advantages (based on early clinical trials) over existing options for patients with severe or life-threatening diseases.

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The Pharma Data

JUNE 29, 2021

Concerns about hypoglycemia and weight gain are known barriers when advancing basal insulin therapy , especially with complex insulin regimens ,” says Julio Rosenstock, Director of the Dallas Diabetes Research Center at Medical City, Dallas, TX, and main author of the study. PY Level 1 (ADA definition: <70 mg/dL <3.9

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Conversations in Drug Development Trends

MAY 1, 2024

This process can be daunting, but understanding how to manage feedback effectively is crucial for developing and ultimately gaining approval for new therapies, especially in oncology clinical trials. Each nation has varying definitions of what qualifies as a pivotal study. Japan, and China.

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