GenScript Life Science
Research Grant Program

What We Do

MOMM Diagnostics develops high-sensitivity rapid diagnostic tests. MOMMs proprietary technology opens previously laboratory-based test for in clinic- and self-testing. Our first test in development is preXclude - a rapid test for preeclampsia, offering unprecedented sensitivity at the point-of-care. Preeclampsia is a significant health problem accounting for a third of obstetric costs (over 2 Billion USD) in the US alone. It affects 2-8% of all pregnancies worldwide and can lead to severe short- and long-term complications for both mother and baby. preXclude delivers immediate information to doctors during a pregnancy check-up helping them optimize treatment and reduce the stress and anxiety for expectant mothers.

How GenScript LSRG Helps

The money granted in the MOMMscript project will enable MOMM Diagnostics to utilise GenScript’s expertise in recombinant antibody development and manufacturing to secure the supply of highly sensitive and specific affinity reagents.

What We Do

The proteins in the AAA+ superfamily of ATPases are often described as molecular machines because they form oligomeric structures that harness chemical energy derived from ATP hydrolysis to perform mechanical works on proteins or nucleic acids. These molecular machines are found in all kingdoms of living organisms, and they have evolved to perform different functions, thereby participating in diverse cell regulation processes. My research is focused on elucidating the structural basis for the functions of these molecular machines, by understanding how their oligomeric structures and biochemical properties are coupled together. Many of the AAA+ proteins in higher organisms have evolved from bacterial ancestors with a significantly expanded ATPase region and form supramolecular complexes in cells. Investigating these megadalton complexes poses challenges, demanding innovative approaches such as gene constructs tailored for effective protein purification.

How GenScript LSRG Helps

I am excited about leveraging support from the LSRG-funded gene synthesis service to significantly advance these investigations.

What We Do

Adeno-Associated Viruses (AAVs) are the leading tool in current gene therapy. However, immune responses to AAVs limit their efficacy, reduces patient eligibility, and constitute a major limitation of their therapeutic application.

In this project, we leverage generative AI-based protein design to explore novel AAV capsids able to evade pre-existing immunity. To test the efficacy of infection of our in-silico designs, and to further train the model, we combined directed evolution of chimeric capsids and de novo capsid design. We then tested these new libraries using high-throughput methods in multiple cellular models, such as HEK293T and HepG2, both in presence and in absence of patient-derived sera. Testing with patient-derived sera reports on the ability of novel AAV capsids to evade pre-existing neutralizing antibodies that would decrease therapeutic efficacy.

By iterating generative modeling and screening with sera from a diverse population, we anticipate the discovery of capsid sequences with high probability of correct folding, high immune-evasion potential and a wide applicability across multiple ethnic groups.

How GenScript LSRG Helps

“Thanks to the LSRG grant, we could expand our repertoire of de novo synthesis to 100 synthetic sequences, and have them all synthesized and shipped to us. An endeavor otherwise not possible for an academic lab.”

“The LSRG grant enabled us to pursue two parallel strategies and effectively de-risk our original approach”

What We Do

Age-related macular degeneration (AMD) is a leading cause of irreversible blindness, projected to affect 288 million people worldwide by 2040 due to the growing ageing population. Neovascular AMD (nAMD), the most severe form, leads to sudden central vision loss, caused by leaky blood vessels that bleed into the retina, causing tissue damage and scarring. The rapid onset and demographic of AMD result in high social and healthcare costs.

Although anti-VEGF therapies have revolutionized nAMD treatment, they involve frequent eye injections every 6-8 weeks that is burdensome and unpleasant for patients and caregivers, significantly impacting their quality of life. Importantly, a significant proportion of eyes with nAMD develop retinal/macular scarring and these eyes respond very poorly to anti-VEGF therapies with respect to preservation of vision.

Ikarovec is developing IKC203V, an AAV-based ocular gene therapy for nAMD. IKC203V encodes two secreted proteins: an Fc-fusion protein to neutralise VEGF, a key driver of neovascularisation and a single-chain variable fragment (ScFv) to neutralise CTGF, a key driver of fibrosis/scarring

The efficacy of the anti-CTGF component will be assessed in vivo using an optimised 2-stage laser CNV subretinal fibrosis model, and screened against other anti-fibrotics.

How GenScript LSRG Helps

We are thrilled to be working with GenScript to advance the development of our gene therapy. We have a long-standing relationship with GenScript in relation to plasmid cloning, and we are excited to try their vector manufacture services. This grant has allowed Ikarovec to expand our in vivo anti-fibrotic evaluation, which in turn will help complete a full data package suitable for clinical development.

What We Do

The main focus of our lab is on antibody discovery and characterization, especially for infectious diseases-associated pathogens, including hypervariable viruses such as influenza virus, SARS-CoV-2 and hepatitis C virus (HCV).

More in detail, the lab is equipped with >15 years of research experience focused on the selection, cloning, characterization and engineering of monoclonal antibodies (mAbs), of human or animal origin, and directed against infectious agents. In particular, the potential of these mAbs is investigated as therapeutic molecules, able to directly neutralize infectious pathogens, or as indirect molecules to be exploited for the development of prophylactic approaches and for the modeling of the humoral immune response. Moreover, their possible engineering in order to improve (i.e., their binding or stability characteristics) or gain new functions (such as in the case of chimeric antigen receptors, CARs), represents a promising and alternative approach for difficult-to-eradicate infectious pathogens. In this context, we are committed also in the field of innovative prophylactic countermeasures for the purpose of improving or developing new vaccines for hypervariable pathogens. In particular, we are investigating the mechanisms of protection elicited by standard of care and next-generation influenza vaccines and adopting different strategies for the development of a vaccine against HCV.

How GenScript LSRG Helps

The LSRG-funded GenScript services will benefit the research of our lab for generating the necessary molecular tools for the assessment of the immune response elicited by our experimental HCV vaccines.

What We Do

The liver’s role in drug metabolism makes it an ideal candidate to use for in vitro drug screening applications. Human liver organoids (HLO) are three