Our Mission
Customized Lipid Nanoparticles (LNPs)
for every nucleic acid-based drug
Is your delivery vehicle bringing your payload to the target tissue(s), obtaining a good level of protein expression among majority of the cells in that tissue,
with a good safety profile?
If not, your delivery vehicle is probably not optimized for your payload, target tissue or route of administration.
Conventional wisdom in the lipid nanoparticle (LNP) field takes a one-lipid-fits-all approach for a variety of applications. However, this often yields undesirable results.
Carcell develops bespoke LNP formulation uniquely tailored for your application. From bench to bedside, our team has deep industry expertise in lipid discovery, formulation development and manufacturing to cater to your unique LNP needs.
What We Do
Lipid licensing & LNP R&D services for your nucleic acid-based drug
"Time is money" is especially true in drug development, and therefore time and cost efficiency is at the heart of our business. Depending on your specific LNP needs, Carcell has a range of solutions for you (hover over the boxes):
Licensing
Licensing only
Carcell has a library of >1000 proprietary ionizable lipids to choose from. These lipids have been tested rigorously in vivo for potency and safety, benchmarking them against some of the lipids found in commercial-stage LNP formulations.
We will work with you to identify the optimal lipid that can be combined with your payload to create your drug candidate. The licensing is typically structured on a "per target" basis.
Co-development & licensing
We can also work with your in-house R&D team to co-develop novel lipids and/or formulation. Some of our in-house formulation capabilities enable:
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Increased potency
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Repeated dosing (PEG-free formulations)
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Tissue-targeting (surface modifications)
Services
Contract Services
Carcell offers an end-to-end suite of services on a fee-for-service (FFS) model:
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Lipid discovery and customized lipid synthesis
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Encapsulation of payloads into LNPs
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LNP composition optimization, and/or formulation process development
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In vivo testing in rodents and non-human primates (NHPs)
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Research- or GMP-grade manufacturing
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Project management at different stages of drug development.
Why Work With Us
We take pride in our LNP capabilities &
bring you tremendous value
We have established a systematic and cost-efficient way of screening novel lipids in animals. - Our time- and cost-savings can be passed on to you.
Our lipids are potent and safe, potentially enabling lower doses and repeat dosing. - Our lipids increase your odds of success in the clinic.
Our lipid library is chemically diverse and structurally unique. - There are no foreseeable freedom-to-operate roadblocks for your products.
We provide you with a one-stop solution including lipid discovery, formulation development and manufacturing so that you can focus on your payload. - We aim to establish a long-term collaboration with you.
We can customize an LNP formulation for you and bring it to gram-scale without compromising quality.
Our Science
What Are LNPs?
Lipid nanoparticles (LNPs) are advanced drug delivery vehicles composed of lipid-based materials, typically ranging in size from 10 to 200 nm. These nanoparticles act as carriers for therapeutic payloads, such as nucleic acids, facilitating their delivery to target tissues in the body while providing the payload protection against drug degradation and enhancing stability in circulation. Depending on the formulation, LNPs can be administered in a variety of ways including intravenous and intramuscular administration.
Ionizable lipids play a crucial role in the functionality and unique characteristics of LNPs. These are lipids that carry a positive charge at low pH due to the presence of amino or guanidino groups in their structure. This positive charge distinguishes ionizable lipids from other lipid components in the LNP formulation and determines the encapsulation of various payloads and cellular uptake of LNPs in various tissues.
LNPs have diverse applications in various medical fields:
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Gene therapy: Deliver RNA, DNA or gene editing tools to modulate gene expression or correct genetic defects
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Vaccine development: Induce immunity towards antigens of infectious diseases or cancers
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Cancer therapy: Deliver anticancer drugs to tumor sites, and transfect immune cells to express signaling receptors to eradicate tumors
Due to their ability to deliver disease-modifying nucleic acid therapies coupled with their potential applications in personalized medicine, LNPs hold great promise for improving therapeutic outcomes and patient well-being.
Our Approach
Problem: Delivering drugs with unoptimized LNPs because LNP discovery is historically an inefficient process
No structure-activity-relationship in lipid design
No correlation between
in vivo and in vitro results
Lack of customized LNP for different payloads & route of administrations (RoAs)
Solution: Our LNP platform that pushes current boundaries and yields customized LNPs for different applications
Highly productive lipid library development
High throughput formulation
& in vivo screening
Customized LNPs for various payloads and RoAs
How: We perform high throughput screening in vivo to find you the right lipid you need
Carcell has a rapidly growing vault of novel and proprietary ionizable lipids. We test the potency and distribution of these lipids in mice and compare them to commercial benchmarks such as ALC-0315, SM-102 and MC3. The top performers are subjected to further scrutiny such as tolerability
studies and freedom-to-operate (FTO) analysis.
>1500
Lipids in our vault
48
Formulations can be screened in vivo in a single day
>200
New lipids synthesized & screened in vivo every month
>5x
More potent than industry benchmarks (top performers)
With multiple shots on goal in our iterative lipid discovery and screening process, depending on your needs, we will find the LNP most suited for your application.
Our LNPs undergo rigorous analytical testing to ensure that the data generated is robust and reproducible. We ensure that our LNPs meet and even exceed industry standards:
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Average molecular weight
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CAD purity of > 95%
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Size of < 100 nm
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Polydispersity Index (PDI) of < 0.2
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Encapsulation rate of > 90%
We also supercharge your LNP with formulation development:
Besides the choice of ionizable lipid, there are many other knobs to turn. Mixing geometry and rate, buffer composition and concentration, temperatures etc. need to be carefully controlled. At Carcell, we combine our library of process development methods with our high throughput LNP formulation instrument to rapidly screen for the optimal production process for your LNP.
Due to our highly cost-efficient R&D, we have the ability to pass our cost-savings to our clients and offer one of the most competitive prices globally.
Working With Us
Customized or off-the-shelf LNPs?
We have you covered!
When developing your bespoke LNP, we will consider all of the following factors:
Target
Tissue
Route of
Administration
Payload/
Application
We also have off-the-shelf LNPs available for licensing, with advanced data for their specific applications:
Gene Editing Liver | IV
Circular RNA Liver | IV
Immune cells Spleen | IV
mRNA Vaccine Muscle | IM
We also provide curated high throughput screening panels for specific applications for finding the best lipid for your needs:
Gene editing tools panel of 12 lipids
Potent liver-targeting panel of 48 lipids
Infectious disease vaccine panel of 48 lipids
Cancer vaccines panel of 12 lipids
Immune cell-targeting panel of 12 lipids
More panels to come
With better lipids discovered every month, we will update these panels to ensure that the best lipids are available for testing. We build specific panels to cater to our partners’ bespoke applications and will expand the number of panels in the time to come. Depending on the specific needs of our partners, we could also encapsulate other payloads such as proteins/peptides and small and large molecules.
Carcell - Your Nucleic Acid Delivery Team
At Carcell, we work closely with our partners to help them achieve their drug delivery goals.
We place emphasis on delivering high quality work to our clients.
Typically, we will establish first contact with an introductory call to better understand the intricacies of your project. Subsequently, we will initiate an evaluation step via a material transfer agreement (MTA) as your first trial of our lipids. Upon successful evaluation, we become partners as we establish an official collaboration, eventually leading to your licensing of our proprietary lipids and/or engaging our services.
First
Contact
Identifying
Your Needs
Evaluation
Phase
Working
Together
We work closely with our partners to make your drug delivery project a reality:
Contact us now for a non-obligatory discussion on your nucleic acid delivery needs!
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All Rights Reserved