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Momentous Cell and Gene Therapies Approved in 2025
03 Nov, 20257+ minutes
Cell and gene therapies (CGTs) are a contemporary class of treatments with a remarkable power to address the needs of rare disease and cancer patients.
Unlike pharmaceuticals and biotechnologies, CGTs are historically very new. With initial concepts and early developments taking place in the 1960s and 70s, it was only in the mid-to-late 2010s that gene and cell therapies became widely considered as the third main drug development platform.
Below we've highlighted three groundbreaking cell and gene therapies that gained FDA approval in 2025. Each fulfills an unmet medical need and will soon provide transformative benefits to patients in the upcoming year.
A brief history of gene and cell therapies
Cell and gene therapies refer to a broader group of medicines, including cell-based immunotherapies, cell therapies, gene therapies and tissue-engineered products. They are defined by using genetic material to alter a patient’s cells for the treatment of rare genetic diseases or cancers.
Although generally grouped together, cell therapies and gene therapies are distinct from one another (although some processes do combine the two). Cell therapy dates back to the 1940s following the first blood transfusion, gaining momentum through organ and bone marrow transplantation in the 60s and 70s. Gene therapy came about in the late 1970s after recombinant DNA technology was discovered.
Since then, significant CGT milestones include the first successful gene therapy in 1990, the development of CAR-T cell immunotherapies in the 2010s and the groundbreaking discovery of CRISPR gene editing technology in 2012 – with the first and only CRISPR therapy becoming approved in 2023.
2025 has seen considerable progress that’s contributed to strong market growth despite the challenge of high costs and regulatory hurdles. Below are three pioneering CGTs that gained FDA approval this year.
Zevaskyn
On April 29th, the FDA approved Abeona’s cell-based gene therapy, Zevaskyn, for recessive dystrophic epidermolysis bullosa (RDEB).
RDEB is a debilitating, rare genetic dermatological condition without a cure. Caused by mutations in the COL7AI gene, people born with this condition cannot sufficiently produce type VII collagen, which is essential for providing the skin with elasticity and strength. As a result, patients suffer with extremely fragile skin that easily blisters from tears and friction. The condition can also severely affect their internal organs.
But now, thanks to Abeona Therapeutics, people living with RDEB have a new source of hope for improving their quality of life. Zevaskyn uses a patient’s own skin cells and surgically applies them to treat wounds and sores. With just one surgical application, patients can expect up to 50% more healing of large, chronic wounds.
The therapy works by delivering a functional copy of the missing COL7AI gene to the patient’s own skin cells. These cells are then expanded into sheets and applied during surgery to open wounds.
The approval of Zevaskyn is a hugely pivotal moment for people struggling with this painful and debilitating condition. With this first-of-its-kind therapy, there now lies opportunity for healing and pain reduction in even the most severe skin wounds.
Encelto
Encelto was approved by the FDA earlier this year on March 6th. The novel eye implant uses cell-based therapy to slow vision loss in rare eye diseases – and is currently FDA approved for the treatment of macular telangiectasia type 2 (MacTel).
MacTel is a rare and slow progressing retinal disease that causes central vision loss due to blood vessels in the macula becoming dilated and leaking. It is usually most noticeable for people in their 50s and 60s, impacting daily activities like reading, driving, recognizing faces – and their overall independence.
Neurotech Pharmaceuticals’ Encelto offers patients a chance to slow the progression of MacTel dramatically, as proven in two Phase 3 clinical studies.
The implant contains 200,000-400,000 allogeneic retinal pigment epithelial (RPE) cells that are modified to produce a ciliary neurotrophic factor (CNTF) protein. Once implanted, the tiny device works by slowing the loss of light-sensing cells and preserving vision in its patients on a long-term basis.
By delaying the onset of the disease with just one surgical application, patients can have a better quality of life without the need for repeated treatments.
Papzimeos
The last CGT to be approved this year is Papzimeos, a groundbreaking gene therapy that can now be used to treat recurrent respiratory papillomatosis (RRP).
RRP varies from patient to patient but can cause severe breathing difficulties that may become life threatening. This condition causes benign tumors (papillomas) to grow anywhere in the air passages but most commonly in the larynx.
Aside from causing voice disorders, frequent surgeries can become necessary to keep airways clear. In severe cases, patients may have to undergo an operation every few weeks.
As no previous medical therapies have eliminated the need for repeated surgeries, the approval of Papzimeos is historic.
Developed by Precigen, who have a cell and gene therapy focus across immuno-oncology, autoimmune disorders and infectious diseases, the immunotherapy is administered via injection. It then causes an immune response against cells infected with the causative agents in RRP.
Papzimeos was given FDA approval following results from a trial including adult patients with RRP who needed three or more surgeries per year. More than half of the patients (51.4%) responded remarkably well – and had no need for any surgical intervention in the 12 months following treatment.
This gene therapy will be transformative for RRP patients, especially those who – until now – have had to revolve their lives around frequent hospitalizations and surgeries.
