Broad Institute

JUNE 11, 2025

Broad Institute and Bayer extend their cardiovascular alliance By Leah Eisenstadt June 11, 2025 Breadcrumb Home Broad Institute and Bayer extend their cardiovascular alliance Partnership will continue with renewed focus on developing new cardiovascular therapies.

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Alta Sciences

JUNE 26, 2025

AI Innovations in Ophthalmic Research ARVO is one of the largest global conferences for ophthalmology research , attracting nearly 11,000 attendees from over 60 countries. Hosted by the Association for Research in Vision and Ophthalmology, it serves as a platform for exchanging cutting-edge research findings.

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Broad Institute

FEBRUARY 27, 2025

An ancient RNA-guided system could simplify delivery of gene editing therapies By Corie Lok February 27, 2025 Breadcrumb Home An ancient RNA-guided system could simplify delivery of gene editing therapies The programmable proteins are compact, modular, and can be directed to modify DNA in human cells.

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Drug Target Review

JUNE 16, 2025

Targeting GPCRs to fight inflammatory diseases GPCR-targeting drugs are well known therapies for a range of disease types, including cardio-metabolic, central nervous system and endocrinological disorders. GPCR-targeting therapies such as DT-9046 represent a compelling opportunity in that respect, with major clinical and commercial potential.

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Conversations in Drug Development Trends

NOVEMBER 5, 2024

In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.

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Broad Institute

MARCH 25, 2025

By Leah Eisenstadt March 25, 2025 HEARD organizers, from left to right, Alycia Halladay (Autism Science Foundation), Jenny Mai Phan (George Mason University), and Celia van der Merwe of the NeuroDevelopmental Variability Initiative at the Broad.

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The Pharma Data

JUNE 16, 2025

This year, WuXi AppTec stood out from more than 1,600 companies across 18 industries in Extel’s 2025 Executive Team Rankings for companies in Asia (with the exception of Japan and Australia/New Zealand). This is demonstrated by its first-place ranking in the Best ESG Program category at Extel’s 2025 Asia Executive Team Awards.

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Drug Target Review

JULY 7, 2025

Persistent challenges with NHP use Despite their biological relevance, using live NHPs in research poses several major challenges: Ethical concerns NHPs, due to their advanced cognition and social behaviour, are at the centre of ongoing ethical debates. Supply chain disruptions Availability is a critical bottleneck in NHP-based research.

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The Pharma Data

JUNE 26, 2025

FDA Approves Streamlined Monitoring Requirements and REMS Program Removal for Bristol Myers Squibb’s CAR T Cell Therapies Breyanzi and Abecma, Marking Milestone Toward Expanding Access to Cancer Treatment In a significant regulatory development, Bristol Myers Squibb announced that the U.S.

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The Pharma Data

JUNE 13, 2025

The data were presented during the 2025 European Hematology Association (EHA) Congress (Abstract #S239). Standard single-target CD19-directed CAR T-cell therapies typically enable long-term remissions in roughly 40% of patients, emphasizing a significant unmet need for new and more effective treatment strategies.

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Broad Institute

MAY 21, 2025

Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders. Ben-Simon et al.) and striatum ( Hunker et al.

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The Pharma Data

JUNE 9, 2025

Xywav: A Low-Sodium Alternative with FDA Approval Xywav is a uniquely formulated, low-sodium oxybate therapy, and remains the only product of its kind approved by the U.S. In parallel, researchers observed a drop in 24-hour urinary sodium excretion, reflecting the decrease in sodium intake achieved by the switch to Xywav.

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Broad Institute

JUNE 16, 2025

By Allessandra DiCorato June 16, 2025 Credit: Allison Colorado, Broad Communications Giulia Monti studies Alzheimer's disease in the lab of Anna Greka. By Allessandra DiCorato June 16, 2025 Credit: Allison Colorado, Broad Communications Giulia Monti studies Alzheimer's disease in the lab of Anna Greka.

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Drugs.com

JANUARY 26, 2025

26, 2025 -- Could a virtual avatar be the future of mental health care? New research shows that artificial intelligence (AI) and virtual reality (VR) goggles could revolutionize therapy -- helping patients overcome addiction and. SUNDAY, Jan.

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The Pharma Data

JUNE 26, 2025

Located in Jiangsu Province, China, the Nantong site serves as a critical component of WuXi AppTec’s expansive Contract Research, Development, and Manufacturing Organization (CRDMO) platform. For instance, the layout fosters natural daylighting, open communication channels, and physical proximity among different research units.

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Drug Target Review

JULY 8, 2025

With a background spanning drug development, clinical strategy and translational research, Eleni brings more than two decades of experience to the role. Not only are they chasing new targets, they are also working to understand the biology behind resistance, discover biomarkers that matter and build smarter therapies from the ground up.

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New Drug Approvals

APRIL 2, 2025

1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] 1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] Suzetrigine CAS 2649467-58-1 Weight Average: 473.4 under nitrogen.

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BioPharma Drive: Drug Pricing

JUNE 23, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. Thus, these drugs best complement, rather than replace, healthy lifestyle interventions.

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Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. This approach limits the transformative potential of the technology.

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The Pharma Data

JULY 3, 2025

Comprehensive Report Reveals Key Trends in Gynecology Collaboration and Licensing Deals from 2016 to 2025 Gynecology Collaboration and Licensing Deals report offers a deep dive into the evolving landscape of partnerships in women’s health, providing unparalleled insights into over 150 strategic deals executed globally between 2016 and 2025.

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The Pharma Data

JUNE 25, 2025

Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.

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The Pharma Data

JUNE 26, 2025

BeOne Medicines Showcases Bold Vision and Expansive Oncology Pipeline at 2025 R&D Day BeOne Medicines Ltd., a global oncology-focused biopharmaceutical company, delivered a comprehensive update to investors today during its highly anticipated Research and Development (R&D) Day. Global Head of R&D. “We

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Drug Target Review

DECEMBER 16, 2024

Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.

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Fierce BioTech

JANUARY 8, 2025

Michael Thurn Promotion Start Fri, 01/10/2025 - 16:00 Promotion End Thu, 07/10/2025 - 16:00 Duration 60 Minutes Amyotrophic Lateral Sclerosis (ALS) remains one of the most complex neurological diseases to understand and treat. Advancing therapies requires innovative science, collaborative efforts, and forward-thinking strategies.

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The Pharma Data

JUNE 18, 2025

The ADA’s annual meeting is one of the most influential forums for showcasing scientific and clinical advancements in diabetes and metabolic disease research. The ADA’s annual meeting is one of the most influential forums for showcasing scientific and clinical advancements in diabetes and metabolic disease research. to 3:00 p.m.

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The Pharma Data

JUNE 6, 2025

The data underscored the significant clinical benefits associated with cystic fibrosis transmembrane conductance regulator (CFTR) modulators—particularly its next-generation triple therapy, ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor).

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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. Visit broad.io/L2C2023

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Drug Target Review

JUNE 5, 2025

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

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Alta Sciences

FEBRUARY 19, 2025

Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development pmjackson Wed, 02/19/2025 - 21:30 The unique physicochemical properties of oligonucleotides require the use of specialized bioanalytical approaches, with key considerations including selectivity and specificity, sensitivity, stability, and matrix effects.

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Alta Sciences

JANUARY 29, 2025

Fast-Track Gene Therapy Discovery With Easy Access to NHPs blussier Wed, 01/29/2025 - 15:57 HTML Speed Up Your Gene Therapy Discovery at Our Sacramento Site Do you need quick, hassle-free access to a large pool of nonhuman primates (NHPs) for your gene therapy discovery work?

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The Pharma Data

JUNE 18, 2025

Pushkal Garg to Lead Unified Research and Development Organization, Signaling Strategic Expansion into Next Phase of RNAi Therapeutics Innovation Alnylam Pharmaceuticals , a global leader in RNA interference (RNAi) therapeutics, has announced the promotion of Pushkal Garg, M.D., He holds a B.A.

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The Pharma Data

JUNE 12, 2025

The data were revealed during an oral presentation (S137) at the 2025 European Hematology Association (EHA) Congress in Vienna, reflecting ongoing progress in developing new treatment options for patients battling this aggressive hematologic malignancy.

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Drug Target Review

JULY 3, 2025

Leveraging the extensive breadth of available data to identify entities and relationships across data sources, clinical research experts, therapeutic specialists, machine learning (ML) engineers and others can collectively evaluate areas of interest that may create new opportunities for the asset and a broader clinical strategy.

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The Pharma Data

JUNE 9, 2025

A Game-Changing Growth Hormone Therapy Lonapegsomatropin is a long-acting recombinant human growth hormone (rhGH) therapy designed to be administered once weekly. VISEN Pharmaceuticals expects to obtain regulatory approval for lonapegsomatropin in China by 2025.

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Drug Target Review

JUNE 16, 2025

1 Continuous advancements in sensitivity and spatial resolution support the role of MSI in pharmaceutical research. By integrating MSI with histopathology, clinical chemistry and DMPK data, researchers can develop mechanistic hypotheses of toxicity, improving decision making and reducing unnecessary follow-up studies. References [1].

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The Pharma Data

JUNE 15, 2025

The data, which encompass up to 3 years of follow-up after a single infusion of the treatment, were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, held from June 13–16 in Glasgow, United Kingdom. President and Chief Executive Officer of Intellia Therapeutics.

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The Pharma Data

JUNE 12, 2025

Foundation, explained during the EULAR 2025 presentation: “IgG4-related disease is a progressive, immune-mediated condition with a significant unmet patient need. This design was meant to enable clinician-researchers to gauge the ability of rilzabrutinib to control disease activity after the withdrawal of standard therapy.

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