CELGENE CORPORATION

The global targeted protein degradation market is projected to grow from USD 0.548 billion in 2023 to USD 4.37 billion by 2034, driven by precision medicine, drug discovery investments, and technological advancements. PROTACs lead the market, with molecular glues expected to grow fastest. Therapy development dominates applications, particularly in cancer and neurodegenerative diseases. Pharmaceutical & biotechnology companies are the largest end-users. North America leads in revenue, while Asia-Pacific shows the highest growth rate.

FDA approved tapinarof cream 1% for atopic dermatitis in ages 2 and older, representing a non-steroidal option with a new mechanism of action. It improves skin barrier and reduces inflammation, showing a remittive effect. Over 80% of participants aged 2-17 in ADORING 3 trial achieved clear or almost clear skin, with 52% reporting completely clear skin.

The global Acute Myeloid Leukemia Therapeutics Market is projected to grow at a CAGR of 13.4% during 2024-2032, driven by advances in targeted therapies and immunotherapies.

Molecular glue degraders, discovered serendipitously, have opened a new field of drug discovery, attracting Big Pharma investments. These small molecules enhance protein destruction by interacting with ligases, targeting 'undruggable' proteins. Major deals in 2024 include collaborations between Biogen, Novartis, Takeda, and Novo Nordisk, potentially worth over $6 billion. The therapeutic potential spans oncology, neurodegeneration, autoimmune, and cardiometabolic diseases. The discovery of cereblon as thalidomide's target in 2010 was pivotal, leading to further research and development in this space.

Gene therapy market to reach $5.3 billion by 2028 with a CAGR of 19.8%. Key players include Novartis AG, Orchard Therapeutics, Celgene, Spark Therapeutics, Gilead Sciences, and Sibiono GeneTech. Market driven by genetic disease prevalence, tech advancements, and R&D investments. Challenges include high costs, manufacturing complexities, ethical concerns, and skill shortages. Future trends include CRISPR adoption, personalized medicine, and non-viral delivery systems.

A review in *Blood Cancer Discovery* outlines a major multiple myeloma research project that supported the FDA's decision to greenlight minimal residual disease (MRD) as a biomarker endpoint for accelerated drug approval, potentially cutting a decade off the drug development process. The research, led by C. Ola Landgren and Sean Devlin, examined how MRD measurements correlate with clinical outcomes, leading to the FDA committee's 12-0 vote in April 2024 to allow MRD as a clinical trial endpoint for accelerated approval in multiple myeloma.

The IDH market is set to grow rapidly due to expanded indications for approved therapies and increased R&D. Key players like Servier and Bayer are developing novel IDH inhibitors, with IDHIFA (enasidenib) being the first-in-class oral targeted inhibitor for IDH2-mutated AML. IDH inhibitors, including TIBSOVO (ivosidenib) and REZLIDA (enasidenib), are FDA and EMA-approved for various cancers. Vorasidenib, in advanced development, aims to treat IDH-mutant diffuse glioma. The market growth is driven by advancements in research and expanding pipeline of IDH inhibitors.

The CEPHEUS trial showed that adding daratumumab (DARA) to bortezomib, lenalidomide, and dexamethasone (VRd) significantly increased minimal residual disease (MRD) negativity and improved progression-free survival (PFS) in transplant-ineligible or deferred patients with newly diagnosed multiple myeloma (NDMM).

The GMMG-HD7 trial evaluated isatuximab (Isa) combined with lenalidomide, bortezomib, and dexamethasone (RVd) in newly diagnosed multiple myeloma patients eligible for autologous stem cell transplantation (ASCT). Results showed significantly higher minimal residual disease negativity (MRDneg) rates with Isa-RVd (66%) compared to RVd alone (48%) after induction therapy, and higher continued MRDneg rates during maintenance therapy. The addition of Isa enhanced MRDneg rates and improved progression-free survival (PFS) outcomes, with MRDneg status being a significant prognostic factor for PFS. The trial demonstrated that Isa-RVd improves MRDneg rates and provides long-term benefits, particularly for patients maintaining MRDneg status throughout treatment.

Stefano Romeo's research identifies two types of steatotic liver disease: one aggressive, affecting the liver, and another linked to cardio-renal-metabolic syndrome. Genetic tests reveal 27 new variants, enabling risk score determination for tailored treatments. Parallel studies using unsupervised clustering confirm these findings, advancing precision medicine.