Looking Back at the Cell and Gene Therapy Leaders That Made Waves in 2024

The cell and gene therapy (CGT) sector was buzzing as 2024 wrapped up, with fresh approvals and promising clinical trial results having lit the way forward. Mesoblast’s Ryoncil snagged FDA approval in December for pediatric graft-versus-host disease, Adaptimmune’s Tecelra got the green light in August for synovial sarcoma, and Casgevy—the pioneering CRISPR-based therapy—scored its first clearance for sickle cell disease in late 2023, followed by a second nod in January 2024 for transfusion-dependent beta-thalassemia (TDT). Big names like Gilead/Kite and Bristol Myers Squibb (BMS) raked in solid quarterly revenues from their CAR-T offerings, while Bluebird Bio, a standout in rare disease treatments, felt the heat from competitors who targeted bigger patient pools with faster market traction. Bluebird’s therapies for ultra-rare conditions started bringing in cash, but giants like Gilead/Kite and BMS gained ground by tapping into broader markets.

The CGT landscape shifted gears, according to the third-quarter 2024 Gene, Cell, + RNA Therapy Landscape Report from ASGCT and Citeline. It showed that 51% of new gene therapy trials focused on non-cancer conditions, marking a pivot from the oncology-heavy past. With 4,099 therapies in development—49% of them gene therapies—the pipeline was packed. Deal activity held steady with 101 transactions in Q3 2024, and early-stage financing nearly doubled from the previous quarter. The contract development and manufacturing organization (CDMO) space saw Agilent snap up Biovectra for $925 million, while global players like ImmunoACT in Mumbai and EdiGene in Beijing pushed new platforms forward. Heavyweights Novartis and Cellectis kept expanding their patent portfolios, cementing their spots as innovation hubs. Here’s a rundown of the top players who shaped this fast-evolving field, broken down by disease area, with a peek at the market vibes that defined each in 2024.

Oncology: A Battle Against Cancer with Precision

Cancer remained a prime target for CGT, with CAR-T therapies having led the charge. Gilead Sciences/Kite Pharma pulled in strong sales—$521 million in Q2 2024 and about $485 million in Q3—thanks to Yescarta and Tecartus. BMS followed close behind, reporting an 8% revenue bump in Q3 2024 to $11.9 billion, with its growth portfolio, including Abecma and Breyanzi, jumping 18% to $5.8 billion. Novartis kept its foothold with Kymriah, while Adaptimmune’s Tecelra tackled synovial sarcoma. Autolus Therapeutics rolled out next-gen CAR-T with Aucatzyl, and Poseida Therapeutics, then under Roche’s wing, advanced non-viral gene editing. The oncology CGT market was red-hot, having been projected to hit $82 billion by 2032, per Precedence Research, driven by rising cancer rates and personalized medicine demand.

Rare Diseases: Targeted the Ultra-Niche

Rare disease therapies carved out a niche, with Bluebird Bio having led the pack. It boasted three FDA-approved treatments—Zynteglo, Skysona, and Lyfgenia—for beta-thalassemia, cerebral adrenoleukodystrophy, and sickle cell disease, respectively. BioMarin’s Roctavian tackled hemophilia A, and Sarepta’s Elevidys targeted Duchenne muscular dystrophy (DMD). Krystal Biotech’s Vyjuvek treated dystrophic epidermolysis bullosa (DEB), while Orchard Therapeutics’ Lenmeldy addressed rare inherited disorders. The rare disease market, valued at $142 billion in 2023 per Fortune Business Insights, was set to grow as therapies expanded into untapped conditions.

Hematologic Disorders: Played with Blood in the Game

Hematologic conditions like sickle cell disease and hemophilia saw breakthroughs. Vertex Pharmaceuticals and CRISPR Therapeutics teamed up for Casgevy, which was approved for sickle cell disease and TDT. Pfizer’s Beqvez targeted hemophilia B, and uniQure’s Hemgenix (with CSL Behring) joined the fray. Mesoblast’s Ryoncil helped with graft-versus-host disease, and Gamida Cell’s Omisirge boosted bone marrow transplants. This market was climbing, having been projected to reach $12 billion by 2030, per Grand View Research, fueled by genetic fixes for blood disorders.

Neuromuscular and Ocular: Kept Nerves and Eyes on the Prize

Neuromuscular and ocular therapies gained traction. Roche’s Luxturna treated inherited retinal disease, pulling in about 30 million CHF in Q3 2024 sales, beating expectations. Astellas Gene Therapies explored neuromuscular and ocular pipelines, while Solid Biosciences focused on DMD. The neuromuscular market sat at $6 billion in 2024, per Statista, with ocular therapies having grown as gene fixes for blindness expanded.

Emerging Platforms: Pushed the Next Big Thing

Innovators pushed boundaries with new tech. Intellia Therapeutics ran the first systemic in vivo CRISPR trial, Beam Therapeutics pioneered base editing, and REGENXBIO’s AAV vectors were widely licensed. ElevateBio’s $401 million Series D backed a CGT innovation hub, and Sana Biotechnology explored hypoimmune platforms. This segment’s market was nascent but booming, with gene editing alone having been eyed at $20 billion by 2030, per Allied Market Research.

What Was Next for CGT?

The CGT field fired on all cylinders in 2024, with a mix of established players and fresh faces having driven progress. From oncology’s blockbuster CAR-Ts to rare disease breakthroughs and cutting-edge editing platforms, the pipeline brimmed with potential.. Patent filings surged, deals multiplied, and the market stood on the brink of significant expansion. As 2025 unfolds, these companies continue to redefine medicine, one therapy at a time, offering a glimpse into what’s next. Keep an eye on trends like scalable manufacturing, broader disease targets, and regulatory shifts—along with standout companies like Bluebird Bio, CRISPR Therapeutics, and Intellia Therapeutics—for clues on where CGT might head this year.

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Author

Bernice Lottering