Best Pharma Innovations of 2024, According to TIME

TIME’s Best Inventions of 2024 list celebrates pharma innovations that are not only scientific marvels but also drivers of accessibility and innovation. Let’s take a look at some of the therapies that earned their spot on this prestigious list, reshaping healthcare for patients worldwide.

Casgevy: A Game-Changer for Beta Thalassemia

CRISPR Therapeutics and Vertex are transforming blood disorder treatment with their gene-editing solution Casgevy (exagamglogene autotemcel [exa-cel]). This one-time gene therapy tackles transfusion-dependent beta-thalassemia (TDT), a disorder causing severe anemia.

TDT patients often rely on frequent transfusions due to impaired hemoglobin. Using CRISPR-Cas9, Casgevy edits a patient’s stem cells to boost fetal hemoglobin (HbF) production, effectively reducing or eliminating transfusions.

Specialized centers handle Casgevy’s administration, where patients receive their edited cells after preparative treatment.

With pediatric trials underway and growing access through 45 global authorized treatment centers (ATCs), Casgevy presents a new option for many previously limited by traditional treatments.

Related: Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

Elevidys: Precision Therapy for Duchenne Muscular Dystrophy

Moving from genetic blood disorders to muscular dystrophy, Elevidys (delandistrogene moxeparvovec-rokl) offers new possibilities for patients.

Elevidys, by Sarepta Therapeutics, is the first gene therapy for Duchenne muscular dystrophy (DMD), designed for patients aged four and older with specific mutations.

Duchenne’s hallmark is the lack of dystrophin, a protein essential for muscle health. Elevidys delivers a modified gene using an adeno-associated virus (AAV) vector, producing micro-dystrophin, which supports muscle function.

Ambulatory patients receive traditional approval, while non-ambulatory cases are covered under accelerated approval, pending further trials.

Elevidys is administered as a single intravenous (IV) infusion at specialized centers, offering a much-needed option for a progressive, life-limiting condition affecting thousands globally.

Imdelltra: Targeted Therapy for Small Cell Lung Cancer

Imdelltra (tarlatamab-dlle) brings a targeted approach to small cell lung cancer (SCLC) treatment.

Developed by Amgen, Imdelltra is the first FDA-approved bispecific T-cell engager (BiTE) therapy for extensive-stage small cell lung cancer (ES-SCLC), an aggressive cancer often resistant to traditional treatments.

Imdelltra works by targeting the DLL3 protein, commonly found on SCLC cells, and redirecting T-cells to attack cancer cells directly. Clinical trials show promising response durations of 9.7 months, with median survival extending to 15.2 months.

Currently, Imdelltra is delivered via infusion, with multiple trials underway to explore combinations with other therapies, aiming to bring durable control and survival benefits to patients facing advanced SCLC.

Cobenfy: A New Class of Treatment for Schizophrenia

Rounding out these breakthroughs, Cobenfy (xanomeline and trospium chloride) brings a pharmacological angle to schizophrenia management.

Cobenfy, by Bristol Myers Squibb,  targets muscarinic receptors — proteins on nerve cells that respond to the neurotransmitter acetylcholine, influencing muscle movement and more — to manage symptoms with fewer side effects. Combining xanomeline, which activates M1 and M4 muscarinic receptors in the brain, with trospium chloride, which limits peripheral side effects, Cobenfy offers a balanced solution for adults with schizophrenia.

Clinical trials in the EMERGENT program showed sustained improvement in symptoms and quality of life, without common antipsychotic side effects like weight gain or movement disorders.

Administered as an oral medication, Cobenfy provides a new option for a condition affecting over 2.8 million Americans.

For each of these therapies, earning a place on TIME’s list adds well-deserved recognition. Each of these drugs goes beyond addressing unmet needs — they represent real, impactful solutions for patients.