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  • Pages
  • Editions
01 Cover
02 Welcome Letter / Sections
03 Article & Interview Directory
04 Section 1: Introduction
05 Introduction to US Life Sciences
06 Janssen Pharmaceuticals Interview
07 Investment Climate
08 MPM Capital Interview
09 Signet Healthcare Partners Interview
10 Xontogeny Interview
11 Insights from EisnerAmper
12 The Life Sciences Regulatory Climate
13 Buchanan Ingersoll & Rooney PC Interview
14 PhRMA Interview
15 PBOA Interview
16 Ernst & Young LLP Interview
17 LaVoieHealthScience Interview
18 Section 2: Introducing the Hubs
19 East Coast
20 BioNJ Interview
21 MassBio Interview
22 Pennsylvania Biotechnology Center Interview
23 West Coast
24 Biocom California Interview
25 QB3-Berkeley Interview
26 Section 3: Drug Discovery and Development
27 Therapeutic Fields On Fire
28 Insmed Interview
29 Mammoth Biosciences Interview
30 Innovations Enhancing the Patient Experience
31 Arcturus Therapeutics Interview
32 Karius Interview
33 Expert Insights: Targeted Therapeutics
34 First Wave BioPharma Interview
35 Aphios Corporation Interview
36 Section 4: Contract Manufacturing and Chemicals
37 A Shifting Landscape
38 Syngene International Interview
39 Cambrex Interview
40 Lubrizol Life Science Interview
41 Aenova Group Interview
42 Keeping Up With Demands
43 Cureline Interview
44 Interview: Murli Krishna Pharma
45 PsychoGenics Interview
46 Expert Insights: Innovative Technologies
47 AMPAC Fine Chemicals Interview
48 TCG Lifesciences Interview
49 CordenPharma Interview
50 Quotient Sciences Interview
51 A Post-Pandemic World
52 New Vision Pharmaceuticals Interview
53 Adare Pharma Solutions Interview
54 Ascendia Pharmaceuticals Interview
55 Chemicals Producers and Distributors
56 Brenntag North America Interview
57 BASF Pharma Solutions Interview
58 Section 5: Technology Services
59 Life Sciences Go Digital
60 RxS Interview
61 Insights from Markem-Imaje
62 AiCure Interview
63 WhizAI Interview
64 Section 6: Company Profiles
65 Brenntag Company Profile
66 Adare Pharma Solutions Company Profile
67 Quotient Sciences Company Profile
68 SK pharmteco Company Profile
69 AiCure Company Profile
70 Markem-Imaje Company Profile
71 TCG Lifesciences Company Profile
72 Murli Krishna Pharma Company Profile
73 Credits

Joseph Payne, President & CEO,

ARCTURUS THERAPEUTICS

"The industry has only scratched the surface of mRNA treatments and is beginning to turn towards targeting hundreds of cell types."

Can you introduce us to Arcturus Therapeutics?

We are a late-stage clinical messenger RNA vaccine and therapeutics company. For vaccines, our self-amplifying RNA, or STARR, addresses dose level issues by considerably reducing dose levels. For therapeutics, we address the challenge of safe and effective delivery of mRNA with LUNAR, our lipid nanoparticle delivery technology that protects and safely transports mRNA molecules to target tissues.

Can you elaborate on how the STARR delivery technology can help create safer, more scalable and more flexible vaccines?

Conventional mRNA vaccines that enter the body through intramuscular injection express an antigen that elicits an immune response. The antigen expression usually lasts for about two to three days before the mRNA molecule degrades. Self-amplifying mRNA, however, has a longer period of expression. This longer expression period likely is crucial for vaccines to allow for significantly reduced dose levels such as the five micrograms per dose that we have achieved for our lead Covid vaccine candidate. Unlike therapeutics, vaccines are meant to be injected into large populations made of healthy individuals. The dose needs to be minimized for safety, as there is a correlation between higher dosages and higher safety risks. For example, Covid-19 vaccines have shown dose-related toxicities such as anaphylactic allergic responses or undesired heart inflammations in rare situations.

Lowering doses also has a benefit from the manufacturing point of view. A factory making five microgram doses can be more efficient than one making 100 microgram doses, thus decreasing manufacturing cost and time. In addition to a lower dose, speed is important during times like a pandemic, and this type of vaccine can be updated frequently to adapt to new disease variants. At Arcturus we have the ability to lyophilize our vaccine products and remove the water component unlike other mRNA vaccines which are shipped as frozen liquids, providing a more stable supply chain and cold chain process.

When do you anticipate Arcturus’ Covid-19 vaccine candidates will enter the market?

In 2021, we advanced ARCT-154, our lead next-generation Covid-19 vaccine candidate. It was rapidly developed and brought into Vietnam where Phases 1 to 3 clinical studies were conducted. We also have a Phase 1/2 booster trial running in Singapore and the US. We are anticipating a final decision on Emergency Use Authorization (EUA) in Vietnam.

In parallel, in the US and Singapore, we are also evaluating our vaccine candidate as a booster. The self-amplifying mRNA is providing consistent results across all variants of interest, including Omicron and the ancestral strain from Wuhan.

To what extent did Covid-19 speed up the development of mRNA-based treatments?

The pandemic created a sense of urgency that accelerated funding and access to resources from governments worldwide. Arcturus received funding from Vietnam and Singapore that enabled us to accelerate our research. Phase 1 of ARCT-154 began in August 2021 in Vietnam and nine months later, we are awaiting a determination for an EUA in Vietnam. Traditionally, drug development has been an 8–12-year process.

This acceleration has had a positive impact on conventional mRNA therapeutics and next-generation RNA technologies. Over the last two years, we moved swiftly from conventional mRNA to self-amplifying mRNA, as opposed to each one taking 10 years to develop and implement. We hope the self-amplifying mRNA will be a more durable vaccine with broader variant coverage, which is desirable as we transition into an endemic booster market.

How can the LUNAR delivery system be used for protein replacement therapies?

Unlike a five-microgram mRNA injection for vaccines, for therapeutics we have to design for systematic administration of larger doses. It is important the mRNA is pure, and the delivery technology is biodegradable. If the delivery vehicles (lipid nanoparticles or LNPs) accumulate, it can pose risks to the liver or lungs, but LUNAR lipids have been shown to degrade after about 48 hours in pre-clinical studies.

We are currently working on a potential treatment for ornithine transcarbamylase (OTC) deficiency, related to the OTC enzyme in the urea cycle that helps process proteins and ensure normal ammonia levels. We can potentially replace the OTC enzyme by delivering its mRNA to liver cells. Once delivered, nature takes over and makes functional OTC enzyme that can potentially cure the disease and may prevent the need for liver transplants. We are also working on an mRNA therapeutic that can be inhaled to access the bronchial epithelial cells to treat cystic fibrosis.

How do you see mRNA-based therapeutics evolving in the coming years?

To have a successful mRNA vaccine or therapeutic, safe delivery is crucial and must be specific to each cell type, of which there are over 200 types in our body. The industry has only scratched the surface of mRNA treatments and is beginning to turn towards targeting those hundreds of cell types.

Next:

Interview: Karius