The FDA announces new Cell and Gene Therapy office

This month, the FDA has announced the creation of a new Cell and Gene Therapy office to manage the number of new candidates coming into the agency. (

Of the 22 cell and gene therapies approved by the FDA, 14 are cord blood-based treatments. The remaining include two gene therapies, seven cell therapies, and five gene modified cell therapies. In total, cell and gene therapies represent just 7% of the 340 approved biologics.

There are currently over 1,000 ongoing clinical trials for cell and gene therapies registered with Of these, the vast majority are in Phase 1 and 2 trials, meaning that they are still far from commercialization, and most will not reach approval. (Source)

Here is a look at some interesting therapies in the approval pipeline.

Atara Biotherapeutics is a leading allogenic T-cell immunotherapy company pioneering the development of transformative therapies for patients with serious diseases by leveraging our novel allogenic Epstein-Barr virus (EBV) T-cell platform.

They currently have a clinical trial Phase 3 ongoing for Tabelecleucel (tab-cel®) for patients with Epstein-Barr virus associated post-transplant lymphoproliferative disease as well as other EBV-associated diseases.  

Treos Bio is a clinical stage company introducing a disruptive computational technology to change the paradigm of cancer immunotherapy development by resolving the dual challenges of patient and tumor heterogeneity. We use proprietary therapeutic peptides, produced through commercially scalable processes without need for tumor biopsy, to stimulate clinically effective cancer-specific immune responses in patients predicted by our diagnostic tests to respond.

Their PEPI Panel  is a unique approach for personalized cancer immunotherapy generation, their Peptide Library (PEPI Panel) currently contains 3,286 proprietary, immunogenic peptide fragments from 184 tumor antigens associated with 19 cancer indications that induce immune responses in the largest subpopulation of subjects, based on HLA genotype (both class I and II) of 16,000 subjects. PEPI Panel approach guides selection of 12 individual peptides tailored to the tumor type and HLA genotype of patients from a warehouse of off-the-shelf peptides permitting rapid, efficient manufacture of a personalized immunotherapy.

Sirnaomics is the first to achieve positive Phase IIa clinical outcomes in oncology clinical-stage RNA therapeutics biopharmaceutical company with a strong presence in both China and the U.S. that is discovering and developing innovative drugs for indications with significant unmet medical needs and large market opportunities. We have built a professional international team for the discovery and development of RNAi therapeutics and mRNA vaccines and therapeutics, based on our proprietary drug delivery technology platforms.

They have a variety of products in the pipeline including STP for basal cell carcinoma.  In addition, they have products in the pipeline for ZSCLC, multiple solid tumors and a pan cancer panel.  They are seeking partnerships for head and neck, bladder and liver cancer.

Istari Oncology – Immune checkpoint inhibitors have revolutionized cancer therapy, but their success is limited by a lack of preexisting anticancer T cells or the inability of anticancer T cells to overcome the immunosuppressive tumor microenvironment.

Lerapolturev is a unique immune activator with multiple mechanisms to stimulate a powerful anticancer response.  It is based on polio virus, the only immune activator to target solid tumors vis CD155.  It also targets key antigen presenting cells (APCs) within the tumor.  They currently have clinical trials for melanoma and bladder cancer.