Celularity, Inc.

Celularity IMPACT Platform

IMPACT, which stands for Immuno-Modulatory Placenta-derived Allogeneic Cell Therapies, is Celularity’s product platform. IMPACT leverages the capabilities of four cell types that are all allogeneic in nature, meaning they can be used in any patient instead of limited only to the patient the cells were derived from, as is common in other cell therapies. The cell types of Celularity’s IMPACT platform include both unmodified and genetically modified natural killer (NK) cells, CAR-T cells, and mesenchymal-like adherent stromal cells (ASCs). The source material of all these cells is the postpartum human placenta, obtained from accredited hospitals and birth centers after proper donor screening and consent. 

NK cells are immune cells with the ability to kill tumor cells and cells infected with a virus. They are equipped with receptors to effectively identify stressed cells for elimination, while simultaneously avoiding the targeting of healthy cells. So far, commercialization of NK cell therapies has been hindered by the ability to effectively scale the production of mature NK cells. However, Celularity’s proprietary process is overcoming these limitations by effectively producing NK cells in just 35 days. 

CAR-T cells are T cells, which are white blood cells that help protect the body from infection and cancer, that have been re-engineered to produce proteins on their surface called chimeric antigen receptors (CARs). CARs are laboratory-created proteins that are designed to bind to specific proteins found on cancer cells. There are currently quite a few CAR-T cell therapies approved in the US for blood cancer treatment, but the current manufacturing process is burdened by high production costs and extended waiting times. However, Celularity’s CAR-T cell therapy is an off-the-shelf product ready for immediate patient use in the necessary effective quantities. 

ASCs possess anti-inflammatory abilities and properties that help regulate the immune system. Unlike current ASC products which are derived from the donor’s own cells and carry the inherited and age-related defects of the donor, placenta-derived ASCs allow for flexible dosing with an off-the-shelf product and likely have superior capabilities because of their fetal origin. Both of Celularity’s two ASC cell therapies, APPL-001 and PDA-002, are in preclinical development.

CYNK-100 for treatment of AML and GBM

CYNK-100 is an unmodified NK cell type being developed for the treatment of acute myeloid leukemia (AML). It is Celularity’s lead platform product, which has completed a phase 1 trial and is now engaged in a follow-up trial. AML accounts for around ⅓ of new leukemia cases in the United States and over 11,500 deaths. CNYK-100 for the treatment of AML has received fast track designation from the FDA and initial trials have shown significant killing against chronic myeloid leukemia cell lines. 

CYNK-100 is also being tested for the treatment of glioblastoma multiforme (GBM), the most aggressive type of brain tumor, which limits diagnosed patients to an average life expectancy of just 14 to 16 months. CYNK-100 has received both orphan drug and fast track designation from the FDA for the treatment of GBM and is currently undergoing testing in a phase 1/2a trial. 

CYNK-101 + mAb for treatment of HER2+ gastric cancer

CYNK-101 is a genetically modified NK cell therapy being developed as a treatment for HER2+ gastric cancers. Human epidermal growth factor receptor 2, or HER2, is a protein that promotes the growth of cancer cells and leads to poor outcomes in many tumors. CYNK-101 treatment is being tested in combination with the monoclonal antibody (mAb), trastuzumab. The program has been granted fast track designation by the FDA and is currently undergoing phase 1/2a trials.

CYCART-19 for treatment of B cell malignancies

CYCART-19 is a CAR-T cell therapy for the treatment of B cell cancers that targets the CD19 receptor, which is a biomarker that is highly expressed in most B cell cancers. Although CYCART-19 demonstrated favorable results in preclinical trials, the FDA has requested additional information from Celularity before planned phase 1 trials of CYCART-19 can commence.

Operational Overview

Celularity has not yet received approval on any cell therapy products and, therefore, has generated no revenue from the sale of placenta-derived cell therapy products. The company has primarily funded operations through the sale of preferred stock and revenues generated from the non-core legacy biobanking and degenerative disease commercial portions of its business. Its biobanking operations consist of the collection, processing, and cryogenic storage of certain birth byproducts on behalf of third parties, while degenerative disease business operations include the manufacture and sale of Biovance and Interfyl. 

Celularity has made several strategic acquisitions over the last few years, including a private umbilical cord blood stem cell and cord tissue bank called LifeBankUSA from Human Longevity Inc, where it also obtained the rights to Biovance and Interfyl. Other notable acquisitions include Anthrogenesis for key cellular therapeutic assets and CariCord, a family cord blood bank. 

Due to the need for additional funding, Celularity entered into a $150 million pre-paid Advance Agreement with Yorkville in September 2022.

Conclusion

Celularity has a long way to go in moving its product candidates toward commercialization. However, Celularity’s IMPACT platform has incredible potential to improve treatment for a broad range of medical needs through its development of inventory-ready, allogeneic cell therapies. Although the biotechnology and cell therapy industry is incredibly competitive, Celularity’s IMPACT platform is protected by a global portfolio of over 1,500 patents.

Celularity is well positioned to maximize commercial gain while increasing human longevity and life quality by providing innovative placenta-derived cell therapies.