Delving into the Latest Updates on Avirmax Biopharma Inc. with Synapse

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Eye Diseases

Congenital Disorders

Cardiovascular Diseases

AAV based gene therapy

Antibody

Disease domain score

A glimpse into the focused therapeutic areas

Technology Platform

Most used technologies in drug development

Targets

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Disease Domain	Count

Endocrinology and Metabolic Disease	2

Top 5 Drug Type	Count

AAV based gene therapy	5
Antibody	1

Top 5 Target	Count

VEGF-A(Vascular endothelial growth factor A)	1

A Phase 1/2a, Open-Label, Multiple-Cohort, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intravitreal (IVT) Injection of ABI-110 (AAV2.N54- VEGF Trap) in Subjects With Neovascular (Wet) Age-Related Macular Degeneration (wAMD), Including Symptomatic Macular Polypoidal Choroidal Vasculopathy (PCV)

This will be a clinical study to assess initial safety and tolerability of IVT ABI-110 in patients diagnosed with wet macular degeneration (wAMD), including symptomatic macular PCV.

Start Date07 Aug 2024

Sponsor / Collaborator

Avirmax Biopharma Inc.

100 Clinical Results associated with Avirmax Biopharma Inc.

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0 Patents (Medical) associated with Avirmax Biopharma Inc.

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7

Literatures (Medical) associated with Avirmax Biopharma Inc.

01 Mar 2024·Molecular Therapy - Methods & Clinical Development

Nonviral delivery of nCas9 for “safe harbor” integration to treat MPS IVA

Author: Ou, Li

01 Jan 2024·Molecular Therapy

Systematic comparison of rAAV vectors manufactured using large-scale suspension cultures of Sf9 and HEK293 cells

Article

Author: Yu, Yanbao ; Peraramelli, Sameera ; Leib, Ryan D ; Li, Ying ; Luo, Ningguang ; Li, Shuyi ; Ou, Li ; Liu, Shengjiang ; Liu, Fang ; Chen, Alan ; Huynh, Derrick ; Dai, Minghua ; Lin, Kevin ; Li, Jinzhong

Recombinant adeno-associated virus (rAAV) vectors could be manufactured by plasmid transfection into human embryonic kidney 293 (HEK293) cells or baculovirus infection of Spodoptera frugiperda (Sf9) insect cells. However, systematic comparisons between these systems using large-scale, high-quality AAV vectors are lacking. rAAV from Sf9 cells (Sf9-rAAV) at 2-50 L and HEK293 cells (HEK-rAAV) at 2-200 L scales were characterized. HEK-rAAV had ∼40-fold lower yields but ∼10-fold more host cell DNA measured by droplet digital PCR and next-generation sequencing, respectively. The electron microscope observed a lower full/empty capsid ratio in HEK-rAAV (70.8%) than Sf9-rAAV (93.2%), while dynamic light scattering and high-performance liquid chromatography analysis showed that HEK-rAAV had more aggregation. Liquid chromatography tandem mass spectrometry identified different post-translational modification profiles between Sf9-rAAV and HEK-rAAV. Furthermore, Sf9-rAAV had a higher tissue culture infectious dose/viral genome than HEK-rAAV, indicating better infectivity. Additionally, Sf9-rAAV achieved higher in vitro transgene expression, as measured by ELISA. Finally, after intravitreal dosing into a mouse laser choroidal neovascularization model, Sf9-rAAV and HEK-rAAV achieved similar efficacy. Overall, this study detected notable differences in the physiochemical characteristics of HEK-rAAV and Sf9-rAAV. However, the in vitro and in vivo biological functions of the rAAV from these systems were highly comparable. Sf9-rAAV may be preferred over HEK293-rAAV for advantages in yields, full/empty ratio, scalability, and cost.

01 Aug 2022·Drug Metabolism and Disposition

Application of Empirical Scalars To Enable Early Prediction of Human Hepatic Clearance Using In Vitro-In Vivo Extrapolation in Drug Discovery: An Evaluation of 173 Drugs

Article

Author: Yan, Zhengyin ; Brown, Suzanne ; Liu, Ning ; Chang, Jae H ; Broccatelli, Fabio ; Kenny, Jane R ; Jones, Robert S ; Leung, Christian

The utilization of in vitro data to predict drug pharmacokinetics (PK) in vivo has been a consistent practice in early drug discovery for decades. However, its success is hampered by mispredictions attributed to uncharacterized biological phenomena/experimental artifacts. Predicted drug clearance (CL) from experimental data (i.e. hepatocyte intrinsic clearance: CL_int, fraction unbound in plasma: f_u,p) is often systematically underpredicted using the well-stirred model (WSM). The objective of this study was to evaluate using empirical scalars in the WSM to correct for CL mispredictions. Drugs (N=28) were used to generate numerical scalars on CL_int (α), and f_u,p (β) to minimize the error (AAFE) for CL predictions. These scalars were validated using an additional dataset (N=28 drugs) and applied to a non-redundant AstraZeneca (AZ) dataset available in the literature (N=117 drugs) for a total of 173 compounds. CL predictions using the WSM were improved for most compounds using an α value of 3.66 (~64%<2-fold) compared to no scaling (~46%<2-fold). Similarly, using a β value of 0.55 or combination of α and β scalars (values of 1.74 and 0.66, respectively) resulted in a similar improvement in predictions (~64%<2-fold and ~65%<2-fold, respectively). For highly bound compounds (f_u,p{less than or equal to}0.01), AAFE was substantially reduced across all scaling methods. Using the β scalar alone or a combination of α and β appeared optimal; and produce larger magnitude corrections for highly-bound compounds. Some drugs are still disproportionally mispredicted, however the improvements in prediction error and simplicity of applying these scalars suggests its utility for early-stage CL predictions. Significance Statement In early drug discovery, prediction of human clearance using in vitro experimental data plays an essential role in triaging compounds prior to in vivo studies. These predictions have been systematically underestimated. Here we introduce empirical scalars calibrated on the extent of plasma protein binding that appear to improve clearance prediction across multiple datasets. This approach can be used in early phases of drug discovery prior to the availability of pre-clinical data for early quantitative predictions of human clearance.

17

News (Medical) associated with Avirmax Biopharma Inc.

28 Nov 2024

·pipelinereview.com

Avirmax Biopharma Announces First Patient Dosed in Clinical Trial of ABI-110, a Groundbreaking Gene Therapy for Wet AMD Including PCV

HAYWARD, CA, USA I November 21, 2024 I Avirmax Biopharma, Inc., a leader in the development of innovative gene therapies, today announced that the first patient has been successfully dosed in the Phase I/IIa clinical trial of ABI-110, the company’s first gene therapy drug for the treatment of Wet Age-related Macular Degeneration (AMD) including Polypoidal Choroidal Vasculopathy (PCV). “We are thrilled to announce this significant milestone in the clinical investigation of ABI-110” said Shawn Liu, Ph.D., Chief Executive Officer of Avirmax Biopharma Inc. “ABI-110 has the potential to revolutionize the treatment landscape for Wet AMD and PCV.” Avirmax doses first patient in Phase I/IIa trial of ABI-110, a pioneering gene therapy for Wet AMD and PCV “Dosing the first patient marks a pivotal step forward in Avirmax’s mission to bring transformative gene therapies to patients suffering from severe retinal diseases.,” said Wendy Murahashi, M.D., Chief Medical Officer of Avirmax Biopharma Inc.. Wet Age-related Macular Degeneration (AMD) and Polypoidal Choroidal Vasculopathy (PCV) are serious retinal diseases that can lead to severe vision loss. Current treatments often require frequent injections and provide only temporary relief. ABI-110 has the potential to offer a more durable and effective solution by addressing the root causes of these conditions at the genetic level. ABI-110, Avirmax Biopharma’s proprietary gene therapy, utilizes an engineered capsid, AAV2.N54, to efficiently deliver therapeutic transgene to the macular retina. This approach aims to provide a long-lasting solution that goes beyond the limitations of current treatments. The Phase 1 clinical trial is designed to evaluate the safety, tolerability, and preliminary efficacy of ABI-110 in patients with Wet AMD and PCV. About Avirmax Biopharma Avirmax Biopharma Inc., based in the San Francisco Bay Area, is a clinical stage biopharmaceutical company specializing in developing next-generation gene therapies for wet AMD, PCV, geographic atrophy (GA), dry AMD, glaucoma and diabetic retinopathy. Utilizing proprietary AAV vector technologies, Avirmax Biopharma aims to deliver safe, effective, and accessible gene therapies to improve patient outcomes and preserve vision. Visit us at https://avirmax.com/biopharma/ for more information. SOURCE: Avirmax

Gene TherapyPhase 1

22 Nov 2024

·pipelinereview.com

Avirmax Biopharma Announces First Patient Dosed in Clinical Trial of ABI-110, a Groundbreaking Gene Therapy for Wet AMD Including PCV

HAYWARD, CA, USA I November 21, 2024 I Avirmax Biopharma, Inc., a leader in the development of innovative gene therapies, today announced that the first patient has been successfully dosed in the Phase I/IIa clinical trial of ABI-110, the company’s first gene therapy drug for the treatment of Wet Age-related Macular Degeneration (AMD) including Polypoidal Choroidal Vasculopathy (PCV). “We are thrilled to announce this significant milestone in the clinical investigation of ABI-110” said Shawn Liu, Ph.D., Chief Executive Officer of Avirmax Biopharma Inc. “ABI-110 has the potential to revolutionize the treatment landscape for Wet AMD and PCV.” Avirmax doses first patient in Phase I/IIa trial of ABI-110, a pioneering gene therapy for Wet AMD and PCV “Dosing the first patient marks a pivotal step forward in Avirmax’s mission to bring transformative gene therapies to patients suffering from severe retinal diseases.,” said Wendy Murahashi, M.D., Chief Medical Officer of Avirmax Biopharma Inc.. Wet Age-related Macular Degeneration (AMD) and Polypoidal Choroidal Vasculopathy (PCV) are serious retinal diseases that can lead to severe vision loss. Current treatments often require frequent injections and provide only temporary relief. ABI-110 has the potential to offer a more durable and effective solution by addressing the root causes of these conditions at the genetic level. ABI-110, Avirmax Biopharma’s proprietary gene therapy, utilizes an engineered capsid, AAV2.N54, to efficiently deliver therapeutic transgene to the macular retina. This approach aims to provide a long-lasting solution that goes beyond the limitations of current treatments. The Phase 1 clinical trial is designed to evaluate the safety, tolerability, and preliminary efficacy of ABI-110 in patients with Wet AMD and PCV. About Avirmax Biopharma Avirmax Biopharma Inc., based in the San Francisco Bay Area, is a clinical stage biopharmaceutical company specializing in developing next-generation gene therapies for wet AMD, PCV, geographic atrophy (GA), dry AMD, glaucoma and diabetic retinopathy. Utilizing proprietary AAV vector technologies, Avirmax Biopharma aims to deliver safe, effective, and accessible gene therapies to improve patient outcomes and preserve vision. Visit us at https://avirmax.com/biopharma/ for more information. SOURCE: Avirmax Biopharma

Gene TherapyPhase 1

22 Nov 2024

·www.drugs.com

First Patient Dosed in Clinical Trial of ABI-110, a Groundbreaking Gene Therapy for Wet AMD Including PCV

HAYWARD, Calif., Nov. 21, 202. Avirmax Biopharma, Inc., a leader in the development of innovative gene therapies, today announced that the first patient has been successfully dosed in the Phase I/IIa clinical trial of ABI-110, the company's first gene therapy drug for the treatment of Wet Age-related Macular Degeneration (AMD) including Polypoidal Choroidal Vasculopathy (PCV). "We are thrilled to announce this significant milestone in the clinical investigation of ABI-110" said Shawn Liu, Ph.D., Chief Executive Officer of Avirmax Biopharma Inc. "ABI-110 has the potential to revolutionize the treatment landscape for Wet AMD and PCV." "Dosing the first patient marks a pivotal step forward in Avirmax's mission to bring transformative gene therapies to patients suffering from severe retinal diseases.," said Wendy Murahashi, M.D., Chief Medical Officer of Avirmax Biopharma Inc.. Wet Age-related Macular Degeneration (AMD) and Polypoidal Choroidal Vasculopathy (PCV) are serious retinal diseases that can lead to severe vision loss. Current treatments often require frequent injections and provide only temporary relief. ABI-110 has the potential to offer a more durable and effective solution by addressing the root causes of these conditions at the genetic level. ABI-110, Avirmax Biopharma's proprietary gene therapy, utilizes an engineered capsid, AAV2.N54, to efficiently deliver therapeutic transgene to the macular retina. This approach aims to provide a long-lasting solution that goes beyond the limitations of current treatments. The Phase 1 clinical trial is designed to evaluate the safety, tolerability, and preliminary efficacy of ABI-110 in patients with Wet AMD and PCV. About Avirmax Biopharma Avirmax Biopharma Inc., based in the San Francisco Bay Area, is a clinical stage biopharmaceutical company specializing in developing next-generation gene therapies for wet AMD, PCV, geographic atrophy (GA), dry AMD, glaucoma and diabetic retinopathy. Utilizing proprietary AAV vector technologies, Avirmax Biopharma aims to deliver safe, effective, and accessible gene therapies to improve patient outcomes and preserve vision. Visit us at https://avirmax.com/biopharma/ for more information. SOURCE Avirmax Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

Gene TherapyPhase 1

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100 Translational Medicine associated with Avirmax Biopharma Inc.

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Pipeline

Pipeline Snapshot as of 05 Feb 2025

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Discovery

2

3

Preclinical

Phase 2 Clinical

1

7

Other

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Current Projects

Drug(Targets)	Indications	Global Highest Phase

ABI-110	Wet age-related macular degeneration More	Phase 2 Clinical
AAV2.N54 Aflibercept ( VEGF-A )	Age Related Macular Degeneration More	Preclinical
ABI-201	dry age-related macular degeneration More	Preclinical
ABI-202	Proliferative retinopathy with diabetes mellitus More	Preclinical
ABI-107	Non-infectious posterior uveitis More	Discovery