FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. The Natural History Studies Grants Program funds natural history studies that collect gather data on how rare diseases progress over time without treatment.

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The Pharma Data

JUNE 25, 2025

Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).

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The Pharma Data

JUNE 16, 2025

a principal investigator for the TRANSCEND FL study and a lymphoma and cell therapy specialist at Memorial Sloan Kettering Cancer Center in New York. This high durability highlights the sustained Benefit provided by liso-cel in a population typically faced with poor prognosis after multiple lines of standard therapy. months, and 24.5

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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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Drug Target Review

JUNE 16, 2025

In this article, Stephan Schann, Chief Scientific Officer at Domain Therapeutics , explains why this class of drug target is so useful despite the challenges they present. GPCR-targeting therapies such as DT-9046 represent a compelling opportunity in that respect, with major clinical and commercial potential.

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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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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

DECEMBER 23, 2024

While these therapies hold great promise for improving cancer treatment outcomes, their development presents significant challenges, especially in achieving the optimal balance between efficacy and safety. Properly managing these toxicities is crucial to enhancing the safety and therapeutic effectiveness of ADC treatments.

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Drug Target Review

JULY 8, 2025

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. mCRPC is a particularly aggressive form of the disease, and patients currently have limited treatment options,” said Eleni. “As

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The Pharma Data

JUNE 18, 2025

The presentations will also include new insights from Amgen’s Phase 3 FOURIER study of Repatha® (evolocumab) and the VESALIUS-REAL study exploring real-world lipid management practices. The findings are expected to inform healthcare systems and providers about adherence gaps and optimization strategies in statin and non-statin therapy use.

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The Pharma Data

JUNE 13, 2025

The data were presented during the 2025 European Hematology Association (EHA) Congress (Abstract #S239). Currently, treatment options for patients with relapsed or refractory disease remain limited, and prognosis is poor. The most frequently encountered form within this group is diffuse large B-cell lymphoma (DLBCL).

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Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.

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The Pharma Data

JUNE 26, 2025

Held at a pivotal juncture for the company, the R&D Day offered an opportunity for BeOne’s executive and scientific leaders to present new clinical data, pipeline milestones, and strategic insights that reaffirm its mission: to build the world’s first next-generation oncology company.

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Drug Target Review

NOVEMBER 8, 2023

Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.

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SugarCone Biotech

MARCH 2, 2025

Where TGF- is present there is no or limited IFN- secretion by T cells and that means no PD-L1 expression within the tumor microenvironment, aka the TME. VEGF is a secreted protein present in solution, but is also bound to VEGF receptor R2-positive endothelial cells within the TME. No chemotherapy regimen is used in the trial.

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PPD

OCTOBER 17, 2024

Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.

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DrugBank

MARCH 12, 2025

  The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. Beyond vaccines, mRNA is being utilized to enhance the efficacy of existing cancer treatments.

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Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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SugarCone Biotech

JUNE 19, 2025

They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis.

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Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. Only three of the CAR-NK studies were for the treatment of solid tumours.

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FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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The Pharma Data

JUNE 9, 2025

The acceptance, which follows the CDE’s grant of Priority Review status in May, represents an accelerated regulatory pathway for what may become the first approved systemic therapy for TGCT in China. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co.,

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Drug Target Review

MARCH 7, 2024

1 Although pancreatic cancer is rarer than the abovementioned cancers, it claims over 50,000 lives each year in the United States because of a lack of effective treatments. Since then, two therapies have been approved for use in lung and breast cancer but it did not advance pancreatic cancer treatment.

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ASPET

OCTOBER 12, 2023

Supramolecular nanostructured based delivery systems are emerging as a meaningful approach in treatment of cancer, offering controlled drug release and improved therapeutic efficacy. Different SNs approaches and recent literature reviews on peptide delivery are also presented to the readers.

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Drug Target Review

AUGUST 31, 2023

Stem cell therapies have already demonstrated their prowess in treating diverse cancers and ailments linked to the blood and immune system. She expressed enthusiasm for the prospect of leveraging hematopoietic stem cell therapy to devise an innovative therapeutic approach for this devastating disorder.

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Chemical Biology and Drug Design

DECEMBER 15, 2023

Due to the complex pathological processes underlying neurodegeneration, at present, there is no viable therapy available for neurodegenerative disorders. Abstract Neurodegenerative disorders, which affect millions worldwide, are marked by a steady decline of neurons that are selectively susceptible.

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Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? Part of the challenge is that patients present with very different kinds of clinical phenotypes, meaning the populations are heterogeneous. All stakeholders have benefited from these innovations.

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The Pharma Data

JUNE 23, 2025

These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. According to Merck, the addition of WINREVAIR to background therapy resulted in a meaningful improvement in patient outcomes, particularly in delaying disease progression and reducing the risk of severe clinical events.

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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. A Scant Protein with Outsize Importance Slowing the muscle decline of DMD presents two huge challenges: the ubiquity of muscle and a giant gene. The gene presents a complex landscape. million DNA bases.

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Drug Target Review

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

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The Pharma Data

JUNE 22, 2025

The study marks a significant step forward in the treatment landscape for haemophilia A, demonstrating not only clinical safety and pharmacokinetic stability, but also strong patient preference for the Mim8 pen-injector delivery system. With new non-factor therapeutic options, many patients may feel uncertain about switching treatments.

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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Drug Target Review

OCTOBER 25, 2023

Their findings may result in new treatments for reproductive conditions. He stated that this could inform new treatments for reproductive conditions like recurrent pregnancy loss and preeclampsia. Preeclampsia may present at any gestation but is more commonly encountered in the third trimester.

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Chemical Biology and Drug Design

NOVEMBER 23, 2023

Nowadays, gene therapy is being implemented for its treatment as several of these genetic defects have been identified. The present article describes the basics regarding the disease, its causes and factors responsible, and the gene therapy solutions available to treat this disease.

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PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

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The Pharma Data

JUNE 6, 2025

At this premier global meeting focused on thrombosis, hemostasis, and bleeding disorders, Sanofi will present compelling data across 18 scientific abstracts, including five oral presentations. In addition, two poster presentations will offer deeper insights into the LUNA 3 trial.

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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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