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.

Research

Drug Target Review

JULY 21, 2025

Current challenges in CNS drug development Drug development for the CNS is particularly challenging and researchers face several hurdles to producing effective and safe treatments, many of which are unique to the CNS. Moreover, regulatory pathways also change considerably based on the type of therapy – and guidance is sparse.

Drug Development

Elrig

MARCH 25, 2025

There will also be a poster award for early-career professionals recognising the research that the next generation of scientists bring to drug discovery. In addition, the conference comprises poster presentations, networking sessions, a vendor exhibition and a tour of AstraZenecas R&D centre.

RNA

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.

Disease

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.

FDA Approval

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.

RNA

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.

Disease

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.

FDA Approval

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.

Drug Research

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.

Therapies

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.

Government

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.

Therapies

Alta Sciences

JULY 31, 2025

By integrating VoxCell’s high-resolution 3D bioprinted tissue platforms into Altasciences’ discovery and preclinical services, both companies aim to reduce R&D timelines and increase the success rate of investigational therapies. Julie-Ann Cabana Altasciences jcabana@altasciences.com Weight 13 Tags Preclinical Research

Drug Development

Fierce BioTech

JULY 11, 2025

Characterizing Antibody-Drug Conjugates Through Innovative Analytical Solutions scox Fri, 07/11/2025 - 10:15 Tue, 08/19/2025 - 11:00 Resource Type Webinar Mihir Thakar Jingwen Ding, PhD Promotion End Fri, 01/16/2026 - 17:21 Antibody-Drug Conjugates (ADCs) are a powerful and promising treatment options for patients.

Drugs

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.

Drugs

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.

Drug Development

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.

RNA

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.

Drug Development

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.

Therapies

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].

Drugs

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.

RNA

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).

Therapies

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.

Treatment

BioPharma Drive: Drug Pricing

JULY 8, 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. Drugmakers are working to coopt this process with targeted therapies. You can unsubscribe at anytime.

Targeted Protein Degradation

Alta Sciences

JULY 22, 2025

45 — Discovery Studies: An Agile Approach to Early Data for Quicker Decisions pmjackson Tue, 07/22/2025 - 21:31 The early identification of toxicity , and flexibility to explore parameters and characteristics of drug candidates in vivo via early discovery non-GLP studies , allow for improved drug development strategy and faster go/no-go decisions.

Virus

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.

Disease

Broad Institute

JULY 21, 2025

Williams July 21, 2025 Credit: Kathleen Sweadner, Christine Simmons, and Alfred George Scientists studied cultured human neurons, shown here under a microscope, that have a protein (green) with a mutation that causes alternating hemiplegia of childhood. The researchers first tested their strategies in cultured cells from patients with AHC.

Disease

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

Clinical Trials

Broad Institute

MAY 21, 2025

Merkin Prize in Biomedical Technology for developing chimeric antigen receptor (CAR) T-cell therapy, a groundbreaking form of personalized cancer immunotherapy that turns T cells into tumor killers and has led to durable remissions in tens of thousands of patients with previously incurable blood cancers.

Therapies

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.

FDA

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.

Drug Development

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.

Disease

Drugs.com

JULY 14, 2025

MONDAY, July 14, 2025 — Good news for women of a certain age: Hormone replacement therapy for menopause appears to boost the effectiveness of GLP-1 weight loss drugs like Wegovy and Zepbound, a new study says. Women using tirzepatide.

Therapies

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.

Laboratories

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.

Treatment

The Pharma Data

JUNE 23, 2025

These pivotal findings were unveiled at the 85th Scientific Sessions of the American Diabetes Association (ADA) and were concurrently published in The New England Journal of Medicine , underscoring the significant promise of MariTide in the growing field of anti-obesity therapies. This perspective was echoed by Dr. Ania Jastreboff , M.D.,

Trials

Pharma Manufacturing

JUNE 17, 2025

Magazine ENEWS SUBSCRIBE WEBINARS PODCASTS EBOOKS ABOUT US Edit submissions Media kit Innovation Awards Articles Flow state: The evolving shape of continuous manufacturing June 17, 2025 While the pharmaceutical industry has been slow to adopt continuous manufacturing, it appears to be gaining momentum.

RNA