Four Companies We’re Pumped To See at This Week's American Society of Gene & Cell Therapy (ASGCT) Meeting
This week’s ASGCT annual meeting in Los Angeles will bring together pioneers from all corners of the gene and cell therapy* world to share scientific breakthroughs, manufacturing successes, regulatory guidance, and more.
The challenging part: How to allocate our time on the floor! Out of necessity, we've narrowed down a hit list that includes the following technologies and platforms from four emerging companies. Here's why we're so excited...
1.???Tune Therapeutics?–– Manipulating genes from a new angle
Launched in 2021, Tune Therapeutics is one of several start-ups advancing the field of epigenomic editing, which focuses on modulating the expression of disease-associated genes without altering the genetic code.
Tune’s TEMPO platform employs DNA-binding proteins to locate and bind to genes of interest. Once bound, specific effector proteins are recruited to modulate gene expression. These effector proteins include those that add or remove local DNA methylation marks, modulate chromatin accessibility, or recruit transcription factors.
Why we’re pumped...
At the heart of Tune’s platform lies the capability to reversibly control how genes behave. This differs from traditional CRISPR-based gene therapy approaches that permanently alter the genetic code, potentially reducing off-target effects.
The ability to modulate gene expression in a tunable manner means that the gene expression can be increased or decreased, improving on the traditional over-expression or total knock-out achieved by available CRISPR-based gene therapies.
The innovative reversibility and tunability of Tune’s epigenomic editing platform are powering the next generation of gene therapies.
Where to find them at ASGCT...?
Dr. Jennifer Kwon, a senior scientist at Tune, is presenting “Transient Delivery of Epigenome Editors Stably Represses PCSK9 and Lowers LDL Cholesterol in Non-Human Primates” on Friday, May 19, from 9 – 9:15 am.
2.????Carisma Therapeutics –– From CAR-Ts to CAR-Ms
?Founded in 2016, Carisma Therapeutics is a clinical-stage cell therapy company developing novel immunotherapies powered by myeloid cells, such as monocytes and macrophages, that are engineered to express tumor-targeting chimeric antigen receptors (CARs).
Carisma’s cell therapies are engineered to induce pro-inflammatory or anti-inflammatory macrophages to target solid tumors.
Why we’re pumped…
All current FDA-approved CAR-based immunotherapies use CAR-T cells to attack forms of blood cancer (lymphomas and myelomas). Conquering so-called solid tumors has proven more difficult, partly because traditional CAR-T therapies struggle to infiltrate the tumor environment. Carisma uses macrophages engineered to express CARs (CAR-M cells) to overcome this. Macrophages are naturally effective at immune cell trafficking and tumor infiltration. CAR-M cells are also less susceptible to the immunosuppressive tumor microenvironment than CAR-T cells, preventing exhaustion and powering long-lasting treatment. ?
Where to find them at ASGCT...
Dr. Michael Klichinsky, Carisma's co-founder and chief scientific officer, is presenting “Beyond T Cells – Development of CAR-M” on Tuesday, May 16, from 10:45 –?11:05 am.
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3.??Form Bio –– Bringing AI into the biopharma space
Form Bio is a software development company that spun out of Colossal Biosciences in 2022. They offer integrated, end-to-end computational solutions for scientists, including their collaborative data management platform. More recently, they’ve launched an AI-powered platform called FORMsightAI that aims to assist scientists in the design of profitable gene and cell therapies.
The FORMsightAI model encompasses two core components, FORMsightAI SIMULATE and FORMsightAI OPTIMIZE, which use plasmid and bioreactor input data to predict packaging efficiency, improve safety, and maximize payloads. When used in parallel, these platforms can reduce downstream time and costs by preventing foreseeable manufacturing obstacles.
Why we’re pumped…
One of the biggest challenges the gene and cell therapy space is confronting is scalability. The biologically active nature of these therapies means they are inherently bespoke and expensive. Improving the efficiency of biomanufacturing using the booming power of AI could help improve the process and increase the impact of many, many different gene and cell therapies.
Where to find them at ASGCT...
Booth 506
4.?MaxCyte?–– Advancing non-viral gene delivery
MaxCyte, Inc. is an established company with over twenty years of experience in electroporation, the process of delivering molecules into cells using a pulse of electricity.
MaxCyte specializes in Flow Electroporation? technology, which allows for scalable delivery of small molecules to cells, including genes encoding CARs for generating adoptive cell therapies and genes encoding viral vector components for generating gene therapies.
Why we’re pumped…
The current crop of FDA-approved gene therapies uses lentiviral or adenovirus-associated vectors (AAVs) for gene delivery. While effective, this approach can trigger immunogenicity and associated adverse events. MaxCyte is working to facilitate the non-viral delivery of gene therapies by offering its state-of-the-art, scalable electroporation technology.
Where to find them at ASGCT...
MaxCyte is hosting an industry-sponsored symposium on Thursday, May 18, from 12 – 1:30 pm. They can also be found at Booth 355.
*What are gene and cell therapies?
Gene therapies modify the genetic code to replace or modify broken genes underlying disease, while cell therapies use transplanted or genetically engineered cells to regenerate tissue, target tumors, or manipulate disease pathways. Often, these two therapies intersect, as gene therapies can be used to genetically manipulate cell therapies prior to treatment.