Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.

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Fierce BioTech

FEBRUARY 27, 2025

Successful Scale-Up Of An Upstream Viral Vector Process Using An Adherent Platform swheeler Thu, 02/27/2025 - 11:40 Thu, 04/10/2025 - 11:00 Resource Type Webinar Brian Gardell Cell and gene therapies offer hope for previously untreatable diseases, with viral vectors currently favored as the primary gene delivery method.

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Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

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H1 Blog

DECEMBER 11, 2024

In this edition, our CEO and co-founder, Ariel Katz, along with Regional VP of Trial Landscape, Ryan Brown, share their top predictions for 2025. Ryan Brown , Regional VP, Trial Landscape Global standards for diversity plans will emerge in 2025. Ariel Katz , CEO & Co-Founder AI will be used to combat rare diseases.

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Fierce BioTech

JANUARY 16, 2025

On Demand Start Date Thu, 03/06/2025 - 13:00 End Date/Time Thu, 03/06/2025 - 12:00 Listing Image CardinalHealth_ListingLogo_250x190.png png Listing Introduction Optimize patient access and overcome barriers with a structured approach to launch planning.

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Vial

DECEMBER 8, 2023

With technological innovation and scientific research propelling the development of new therapies, anticipation for the future is high. As we look forward to 2024, several groundbreaking therapies are poised to radically change the landscape of ophthalmology, providing hope and improved treatment options for patients with ocular conditions.

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Fierce BioTech

APRIL 8, 2025

Rethinking Drug Commercialization: Navigating a Shifting Biotech Investment Landscape swheeler Tue, 04/08/2025 - 16:40 Thu, 05/15/2025 - 12:00 Resource Type Webinar Susanne Munksted The biotech dealmaking landscape is changingdeals are happening later in development, and securing capital is tougher than ever.

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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. recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Join the club.

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Fierce BioTech

JANUARY 8, 2025

Advancing therapies requires innovative science, collaborative efforts, and forward-thinking strategies. Hear directly from leaders at Ncardia and Neurizon Therapeutics as they share exclusive insights into the latest scientific progress shaping the future of ALS therapies. On Demand Start Date Thu, 02/20/2025 - 14:00

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Codon

SEPTEMBER 29, 2024

The earliest ones relied on simple linear regression and attempted to correlate genetic variations with observable traits or disease risks — such as drug metabolization rates or cancer susceptibility. The 2025 genetic network paper fit that bill exactly. 2024 Statistical models of organisms have existed for decades.

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Drug Target Review

DECEMBER 28, 2023

In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. However, if the therapy targets only the subclonal (branch) mutations, this will result in the mere ‘pruning’ of specific branches rather than elimination of the whole cancer.

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The Pharma Data

JULY 27, 2021

AbbVie (NYSE: ABBV) and Calico Life Sciences today announced an extension of their leading-edge collaboration to discover, develop and bring to market new therapies for patients with age-related diseases, including neurodegeneration and cancer. This is the second extension of the collaboration originally established in 2014.

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Drug Target Review

JULY 17, 2024

How does GigaGen’s single-cell discovery and development platform differ from traditional methods of producing polyclonal antibody therapies? Current methods for producing pAb therapies rely on regular plasma donations for their development. However, recombinant pAbs do not rely on continuous plasma sources for production.

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National Institute on Drug Abuse: Nora's Blog

MARCH 6, 2025

Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.& Retrieved March 6, 2025, [link] U.S.

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Agency IQ

JUNE 21, 2024

CBER’s Peter Marks offers insights on a new Rare Disease Hub, upcoming accelerated approval guidance This week, PETER MARKS, director of FDA’s Center for Biologics Evaluation and Research (CBER), spoke at several sessions of the Drug Information Association (DIA) annual meeting.

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The Pharma Data

SEPTEMBER 16, 2020

SLB linked to 2025 Patient Access Targets to increase patients reached in low- and middle-income countries (LMICs) with strategic innovative therapies by 200% and the Novartis Flagship Programs by 50%. Novartis is taking bold steps to increase patient reach in LMICs with its strategic innovative therapies by at least 200% by 2025.

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The Pharma Data

NOVEMBER 10, 2020

P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease. Crohn’s Disease Therapeutics Market Size $4.7 Billion by 2025. billion by 2025, according to Grand View Research 1. NEW YORK and LONDON, Nov. NEW YORK and LONDON, Nov.

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The Pharma Data

JULY 15, 2021

Trial will include participants at risk for cognitive and functional decline related to Alzheimer’s disease. TRAILBLAZER-ALZ 3 will evaluate whether treatment with donanemab can slow the clinical progression of Alzheimer’s disease in trial participants. vice president of pain and neurodegeneration, Lilly.

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KIF1A

AUGUST 28, 2024

2025 Conference Interest Survey : Help shape the future of our community by sharing your interest in the upcoming 2025 conference. She is the 2nd KAND Pioneer blazing the trail with ASO therapy. 2025 KAND Conference Interest Survey Call all KAND Community Stakeholders! KAND families, please only complete one survey.

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Alta Sciences

JANUARY 28, 2025

Immunogenicity Data Delivered Without Delay blussier Tue, 01/28/2025 - 20:11 HTML Preclinical Assays for Immunogenicity Testing in Clinical Trials Whether developing a vaccine, biologic, or gene therapy, immunogenicity testing is required for regulatory approval and is critical for determining the safety and efficacy of your product.

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The Pharma Data

JUNE 18, 2021

The ADVANCE study included patients with past intolerance or inadequate response to conventional therapy (non-bio-IR) and/or biologic therapy (bio-IR). a Clinical remission per CDAI (Crohn’s Disease Activity Index) is defined as CDAI score of <150. NORTH CHICAGO, Ill. , and daily AP score of ?1,

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Drug Target Review

JULY 1, 2024

As a non-NSAID, it reduces adverse effects associated with these pain relief drugs, including kidney injury, GI gastritis/ulceration and blood pressure elevation, which can affect patients with cardiovascular disease and the elderly. was founded in 2016 to develop safer, non-opioid therapies for acute and chronic pain.

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The Pharma Data

SEPTEMBER 15, 2020

27 key programs highlighted, including assets that could potentially contribute revenue by 2025 and others in the 2026-2028 time frame. Major revenue contributions through 2025 anticipated from Oncology, Vaccines, Rare Disease and Inflammation and Immunology. BNT162 mRNA-based Vaccine Program.

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The Pharma Data

FEBRUARY 22, 2022

Pfizer’s 2025 Opportunity Parity goals represent the company’s commitment to increasing representation of women and minorities at senior -level positions of the organization. Progress made from June 2019 to December 2021 towards different aspects of its 2025 Opportunity Parity Goals are highlighted below.

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The Pharma Data

JUNE 18, 2021

1 Clinical remission was defined by Crohn’s Disease Activity Index (CDAI) in the U.S. “These results represent another step towards the development of risankizumab for these patients, many of whom do not find sufficient disease control with current treatments.” NORTH CHICAGO, Ill. vice chairman and president, AbbVie.

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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

DECEMBER 14, 2022

Digital health products are widely used for the prevention, diagnosis, treatment, and management of health and disease. The introduction of digital medicines—and, more recently, digital therapeutics (DTx)—has begun to influence the treatment and management of certain diseases, both by consumers and by clinicians.

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The Pharma Data

NOVEMBER 16, 2020

In 2020, Regeneron has reinforced our commitment to ‘doing well by doing good,’ as colleagues across the company have worked tirelessly to discover and develop a potential antibody therapy for COVID-19, while at the same time responding to the needs of our colleagues, patients and communities during this public health crisis.”

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The Pharma Data

DECEMBER 12, 2020

Dedicated rare disease unit to be headquartered in Boston. Double-digit revenue growth through 2025; acquisition strengthens AstraZeneca’s broad-based revenue and the company will further globalise Alexion’s portfolio . Geographical presence to be enhanced with broad coverage across primary, speciality and highly specialised care.

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Conversations in Drug Development Trends

MAY 23, 2024

The Critical EU-CTR Transition Deadline The three-year transition period for clinical trials authorized under the CTD that are expected to continue beyond the critical deadline of 30 January 2025 are required to transition to the new EU-CTR via a transition application through the CTIS. However, they also bring unique regulatory challenges.

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LifeSciVC

AUGUST 21, 2024

At this point of the year, the JPMorgan conference seems like ancient history and you are looking into flights for JPM 2025. This is especially true for rare disease trials with so few patients or trials involving patients with numerous concomitant or life-threatening conditions. These goals were believable and achievable.

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The Pharma Data

JUNE 18, 2021

First approval of a conjugate vaccine that helps protect against 20 serotypes responsible for the majority of invasive pneumococcal disease and pneumonia, 1,2,3,4,5,6,7 including seven responsible for 40% of pneumococcal disease cases and deaths in the U.S. Following today’s FDA approval, the U.S. Jansen, Ph.D.,

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The Pharma Data

JUNE 11, 2023

Obesity is one of many cardio-renal-metabolic diseases, which together represent one of the fastest growing health challenges worldwide. With our longstanding heritage in cardio-renal-metabolic diseases, we are excited by the findings and potential implications for millions of people who urgently need healthcare solutions.” “We

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The Pharma Data

JANUARY 12, 2021

For almost 30 years, Alexion has been committed to transforming the lives of those impacted by rare diseases and devastating conditions, and the company remains focused on advancing its innovative therapies and pipeline to drive value for patients and shareholders once we are part of AstraZeneca.”. Chief Executive Officer at Alexion.

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The Pharma Data

JANUARY 15, 2021

“Patients with metastatic HER2 positive gastric cancer with progression following first-line treatment have historically faced poor outcomes, including low response to treatment and rapid disease progression,” said Ronan Kelly, MD, MBA, Director of the Charles A. mg/kg, interstitial lung disease occurred in 10% of patients. Caruth, Jr.

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The Pharma Data

AUGUST 3, 2021

The company’s success is based on its rich R&D portfolio with a focus on diseases with high unmet medical need. This pipeline has the potential to deliver up to 15 medicines for approval by 2025. More than 60 million patients worldwide suffer from this disease, approx.

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The Pharma Data

JANUARY 19, 2021

TCR 2 is developing novel T-cell therapies for solid tumors and hematological cancers. The company is using its pioneering FasTCAR and TruUCAR technology platforms to discover and develop breakthrough cell therapies. The company is based in Suzhou and Shanghai, China. Ortho Clinical Diagnostics is a leader in in vitro diagnostics.

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