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AviadoBio Announces FDA IND Clearance and Fast Track Designation for AVB-101 for the Treatment of Frontotemporal Dementia with Progranulin (GRN) Mutations

By AviadoBio, Press Release, Private Companies
Press Release.

 

AviadoBio Announces FDA IND Clearance and Fast Track Designation for AVB-101 for the Treatment of Frontotemporal Dementia with Progranulin (GRN) Mutations

• AVB-101 is an investigational gene therapy delivered as a one-time infusion directly to the brain, enabling distribution to brain areas affected by FTD while limiting systemic exposure

• The Phase 1/2 ASPIRE-FTD global clinical trial is currently open in Europe and will open in the U.S. in 2024

• FTD is a leading cause of dementia in people under the age of 65

London, UK; November 6, 2023 — AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AVB-101 to be studied in people with frontotemporal dementia (FTD) with mutations in the progranulin (GRN) gene. In addition, the FDA has granted Fast Track designation to AVB-101 for the treatment of FTD-GRN to slow disease progression. Fast Track is a process designed to facilitate the development and expedite the review of new drugs to treat serious conditions and fill an unmet medical need. Fast Track designation is intended to bring new medicines to patients earlier.

AVB-101 has been designed as a potential one-time therapy to halt disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to areas of the brain affected by FTD. Both the U.S. FDA and the European Commission (EC) granted orphan designation to AVB-101 for the treatment of FTD in 2022. The company recently announced enrollment is open in European countries for ASPIRE-FTD, an open-label, multi-center dose-escalation study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN.

“The IND clearance and Fast Track designation of AVB-101 are important milestones for the FTD community and for our company. Sadly, there are currently no disease-modifying therapies approved for the treatment of people living with frontotemporal dementia with progranulin mutations. Receiving Fast Track designation underscores the significant need for treatment options for these patients, and we are eager to soon open U.S. clinical trial sites and offer an innovative option for eligible patients with FTD-GRN,” said Lisa Deschamps, Chief Executive Officer.

“We are using adeno-associated virus (AAV) technology and taking a novel approach to deliver a functional copy of the GRN gene directly to the brain via bilateral, MRI-guided intrathalamic infusion with the aim to restore normal levels of the progranulin protein,” said David Cooper, M.D., Chief Medical Officer. “Our preclinical program shows robust biodistribution to the brain areas where it is needed to restore levels of progranulin, potentially slowing or stopping the progression of FTD-GRN, with little to no progranulin in the rest of the body where it may have adverse effects.”

FTD is a devastating form of early-onset dementia that typically leads to death within seven to 13 years of symptom onset and three to 10 years from diagnosis.¹,² People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.³ FTD is a leading cause of dementia in people under the age of 65⁴ with an estimated prevalence at any one time of up to 4.6 cases per 1,000 of people.⁵ Genetic FTD cases account for about one-third of cases and are most frequently associated with autosomal dominant mutations in three genes, including the progranulin gene.⁶

 

1. Onyike CU. Neuroepidemiology. 2011;37:166–167;
2. Riedl L et al. Neuropsychiatr Dis Treat. 2014;10:297–310;
3. Pressman P and Miller BL. Biol Psychiatry. 2014;75(7):574–581;
4. Hendriks S, Peetoom K, Bakker C, et al. Global Prevalence of Young-Onset Dementia: A Systematic Review and Meta-analysis. JAMA Neurol. 2021;78(9):1080–1090. doi:10.1001/jamaneurol.2021.216;
5. Hogan DB et al. Can J Neurol Sci. 2016;43 (Suppl 1):S96–S109;
6. Young JJ et al. Ther Adv Psychopharmacol. 2018;8(1):33–48.

ABOUT ASPIRE-FTD

ASPIRE-FTD is an open-label, multi-center study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, AVB-101 will be delivered as a one-time treatment into the thalamus via a stereotactic neurosurgical procedure at expert neurosurgical centers throughout Europe and the United States.

More information about the ASPIRE-FTD study can be found at https://clinicaltrials.gov/study/NCT06064890.

 

ABOUT AVB-101

An experimental gene therapy, AVB-101, contains a correct (non-mutated) version of the GRN gene. It is designed to restore levels of progranulin in the brain, potentially slowing or stopping the progression of FTD-GRN. AVB-101 will be delivered as a ‘one-time dose’ directly into the brain via a minimally invasive surgical procedure, performed by a study neurosurgeon at a specialist neurosurgical center.

 

ABOUT AVIADOBIO

AviadoBio’s mission is to develop and deliver potentially transformative gene therapies for people living with devastating neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working relentlessly to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the Company for success in bringing transformative medicines to patients.

AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Lifesciences, and LifeArc.

For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio.

CONTACT

FOR MEDIA ENQUIRIES

Farah Speer
SVP, Head of Communications and External Relations
Fspeer@aviadobio.com
+1-312-543-2881

Jenna Kane
Health+Commerce
jennakane@healthandcommerce.com
+1-480-388-9587

AviadoBio Announces Initiation of Phase 1/2 Clinical Trial

By AviadoBio, Press Release, Private Companies
Press Release.

 

AviadoBio Announces Initiation of Phase 1/2 Clinical Trial, ASPIRE-FTD,

Evaluating AVB-101 in People with Frontotemporal Dementia with GRN Mutations

 

  • AVB-101 is an investigational gene therapy designed to halt disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels in the brain

 

  • AVB-101 is delivered as a one-time treatment into the thalamus to facilitate targeted biodistribution in order to reach broad areas of the brain affected by FTD, while limiting treatment exposure to only the brain itself

 

  • FTD is a leading cause of dementia in people under the age of 65

 

London, UK; October 10, 2023 — AviadoBio, a pioneering gene therapy company developing and delivering transformative medicines for neurodegenerative disorders, today announced that it has initiated the Phase 1/2 ASPIRE-FTD clinical study of its investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with mutations in the progranulin (GRN) gene.

 

ASPIRE-FTD is an open-label, multi-center study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, AVB-101 will be delivered as a one-time treatment into the thalamus via a stereotactic neurosurgical procedure at expert neurosurgical centers throughout Europe and the United States.

 

“FTD is a devastating form of early-onset dementia and there are currently no disease-modifying treatments available,” said Lisa Deschamps, CEO. “The initiation of our company’s first clinical trial demonstrates our commitment to developing and delivering potential transformative gene therapies for those living with neurodegenerative disorders such as FTD.”

 

“While it is known that progranulin protein supplementation is possible using gene therapy, effective brain distribution remains challenging primarily because of the brain’s anatomy,” said David Cooper, M.D., Chief Medical Officer. “Our aim is to use intrathalamic delivery to facilitate biodistribution of the PGRN protein to the areas of the cortex impacted by FTD. AVB-101 has already shown great promise in preclinical studies, and we now look forward to this important next step in its clinical development.”

 

Preclinical safety and biodistribution data following intrathalamic delivery to non-human primates demonstrate good tolerability and widespread progranulin expression in brain tissues. These data will be presented at the 30th Annual European Society of Gene & Cell Therapy (ESGCT) meeting in Brussels, Belgium Oct. 24-27, 2023.

 

People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein. AVB-101 has been designed as a potential one-time therapy to halt disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to the affected areas of the brain.

 

AVB-101 is delivered using a minimally-invasive, stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus has extensive connections to other parts of the brain, including the frontal and temporal lobes, which play a critical role in FTD and the symptoms that impact patients and their families. This targeted delivery method aims to limit the treatment to the brain where it is needed most, thereby reducing the dose required and the potential systemic exposure.

 

More information about the ASPIRE-FTD study can be found at https://clinicaltrials.gov/study/NCT06064890.

 

# # #

 

About Frontotemporal Dementia (FTD) and FTD with GRN mutations (FTD-GRN)

FTD is a devastating form of early-onset dementia that typically leads to death within 7 to 13 years of symptom onset and 3 to 10 years from diagnosis. People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.

 

FTD is a leading cause of dementia in people under the age of 65 with an estimated prevalence at any one time of up to 4.6 cases per 1,000 of people. FTD typically strikes younger than Alzheimer’s disease and the majority of FTD cases occur between 45 and 68 years of age. Given the early onset, FTD has a substantially greater impact on work, family, and finances than Alzheimer’s disease. Genetic FTD cases account for about one-third of cases and are associated with autosomal dominant mutations in three genes, including the GRN (progranulin) gene. Approximately 11,000 people in the U.S. and EU5 are living with FTD-GRN with approximately 2,200 new cases per year. Some FTD cases may be misidentified, and diagnostic delay is common. As disease education, genetics knowledge, and research and treatment options grow, these numbers are expected to increase.

 

About AviadoBio

AviadoBio’s mission is to develop and deliver potentially transformative gene therapies for people living with devastating neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working relentlessly to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the Company for success in bringing transformative medicines to patients.

 

AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Lifesciences, and LifeArc.

 

For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio.

 

Media Contacts:

Farah Speer

SVP, Head of Communications and External Relations

Fspeer@aviadobio.com

+1-312-543-2881

 

Jenna Kane

Health+Commerce

jennakane@healthandcommerce.com

+1-480-388-9587

AviadoBio selected by Fierce Biotech as a ‘Fierce 15’ 2022 winner

By AviadoBio, Press Release, Private Companies
Press Release.

 

AviadoBio selected by Fierce Biotech as a ‘Fierce 15’ 2022 winner

Award reinforces AviadoBio’s position as one of the most promising early-stage biotech companies in the gene therapy industry

London, UK; Monday, September 12, 2022 — AviadoBio, a pioneering, pre-clinical stage gene therapy company focused on developing and delivering transformative medicines for people with neurodegenerative disorders, today announced that it has been selected by Fierce Biotech as a ‘Fierce 15’ 2022 winner, designating it one of the most promising early-stage biotechnology companies in the industry.

The award validates AviadoBio’s progress over the past year and recognizes the mission of the company, and its aspiration to develop gene therapy treatments for patients living with neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) who currently have few-to-no therapeutic options.

“This award acknowledges the immense progress AviadoBio has made this year, and is a testament to the hard work of everyone at the company,”

said Lisa Deschamps, Chief Executive Officer.

“This year has marked some important milestones, including securing orphan drug designation from the FDA and EMA, as we move AVB-101, our investigational AAV gene supplementation therapy AVB-101 for frontotemporal dementia (FTD), into the clinic in late 2022.”

The Fierce 15 celebrates biotech companies who champion innovation and creativity. Every year Fierce Biotech evaluates hundreds of early-stage companies from around the world for its annual Fierce 15 list, which is based on a variety of factors such as the strength of technology, partnerships, venture backers and a competitive market position.

 

About Aviadobio

At AviadoBio, our mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute. AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery. AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, Advent Life Sciences, EQT Lifesciences, Dementia Discovery Fund (DDF), F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), and LifeArc.

The company is developing AVB-101 for patients with FTD-GRN. AVB-101 is an investigational AAV gene therapy designed to slow or stop disease progression by delivering a functional copy of the GRN gene throughout the central nervous system to restore progranulin levels.

For more information, please visit www.aviadobio.com and follow us at Twitter @AviadoBio and LinkedIn AviadoBio.

AviadoBio to Present Pre-clinical Data on its Gene Therapy Candidate for Frontotemporal Dementia at ASGCT 2022

By AviadoBio, Press Release, Private Companies
Press Release.

 

  • Intrathalamic delivery of AVB-101 shows promising efficacy in a pre-clinical disease model
  • Gene therapy candidate also demonstrated broad cortical expression in a large animal biodistribution study

 

London, UK, May 4th, 2022 – AviadoBio, a pioneering, pre-clinical stage gene therapy company focused on developing and delivering transformative medicines for people with neurodegenerative disorders, announces that it will be presenting preclinical data at the upcoming ASGCT 25th Annual Meeting (American Society of Gene & Cell Therapy) on 16-19 May, 2022. The data are from studies it has conducted with its investigational, one-time, adeno-associated virus (AAV) gene therapy, AVB-101 (AVB-PGRN), for the treatment of frontotemporal dementia with GRN mutations (FTD-GRN).

The oral presentation, entitled “Intrathalamic Delivery of AVB.PGRN Rescues Pathology in Grn Null Mice and Achieves Widespread Cortical Expression in a Large Animal Model without Expression in the Liver”, will be presented by AviadoBio Co-Founder and Chief Scientific and Clinical Advisor, Professor Chris Shaw, on Tuesday 17 May at 3:45pm (EST). The presentation will form part of the session “Applications of Improved Gene Therapy Methods in Neurologic Disorders” and will be followed by a Q&A discussion.

The abstract number 468 can be viewed here.

In addition, AviadoBio CEO Lisa Deschamps will be presenting at the conference, on Monday 16 May at 12:00pm (EST), in a session entitled: “Startup Showcase: AviadoBio – A revolution in gene therapy for neurodegenerative disorders”.

ABOUT AVIADOBIO

At AviadoBio, our mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute. AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery. AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, Advent Life Sciences, EQT Lifesciences, Dementia Discovery Fund (DDF), F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), and LifeArc.

The company is developing AVB-101 for patients with FTD-GRN. AVB-101 is an investigational AAV gene therapy designed to slow or stop disease progression by delivering a functional copy of the GRN gene throughout the central nervous system to restore progranulin levels.

For more information, please visit www.aviadobio.com and follow us at Twitter @AviadoBio and LinkedIn AviadoBio.

About Frontotemporal Dementia and AVB-101

Frontotemporal dementia (FTD) is of the second most common form of dementia in people under the age of 65 after Alzheimer’s disease. It affects 50,000 to 60,000 patients in the U.S. and over 100,000 in the E.U. Approximately one third of FTD cases are familial and linked to autosomal dominant mutations in three genes including the granulin gene (GRN) and FTD-GRN represents 5-10% of all patients with FTD. Progressive degeneration of the frontal and temporal lobes of the brain is characteristic of FTD, and is associated with progressive decline of behaviour, decision-making, language and emotion, typically leading to death within 7-10 years of diagnosis. There are currently no approved treatments to stop or slow the progression of FTD or FTD-GRN.

References:

  • Boxer AL. Miller BL.  Alzheimer Dis Assoc Disord. 2005;19 Suppl 1:S3-6
  • Hogan DB, et al. Can J Neurol Sci. 2016;43 Suppl 1:S96-S109
  • Olney NT, et al. Neurol Clin. 2017;35(2): 339–374
  • Greaves CV, et al. J Neurol. 2019;266(8):2075–2086

CONTACT

For media enquiries:
Consilium Strategic Communications
Chris Gardner, Angela Gray, Isabelle Abdou
+44 (0) 20 3709 5700
AviadoBio@consilium-comms.com

AviadoBio™ Raises $80 million in Series A Financing to Advance Neurodegenerative Gene Therapy Platform

By AviadoBio, Press Release, Private Companies
Press Release.

 

  • Series A led by New Enterprise Associates (NEA) and co-led by Monograph Capital follows seeding by Advent Life Sciences, Dementia Discovery Fund (DDF), F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), and LifeArc
  • AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery
  • Co-founded by leading neurologist and neuroscientist Professor Christopher Shaw, molecular neurobiologist Dr Youn Bok Lee and vector biologist Dr Do Young Lee from King’s College London and the UK Dementia Research Institute
  • Lisa Deschamps appointed Chief Executive Officer, bringing 25 years of industry and extensive gene therapy experience

London, UK, December 2, 2021 — AviadoBio Ltd, a pioneering gene therapy company focused on developing and delivering transformative medicines for people living with neurodegenerative disorders, announced today the successful completion of an $80 million (£58.6 million) Series A financing round, following an initial $16.5 million (£12 million) seed financing. The funds will be used to advance AviadoBio’s lead program in frontotemporal dementia (FTD) into the clinic, progress its preclinical assets, including for amyotrophic lateral sclerosis (ALS), whilst continuing to expand its industry-leading team.

The financing was led by New Enterprise Associates (NEA) and co-led by Monograph Capital, with participation from LSP, as well as seed investors Advent Life Sciences, Dementia Discovery Fund (DDF), F-Prime Capital, JJDCand medical research charity LifeArc.

AviadoBio’s technology was developed in the laboratory of Christopher Shaw, Professor of Neurology and Neurogenetics at King’s College London’s Institute of Psychiatry, Psychology & Neuroscience, with the support of the UK Dementia Research Institute, the My Name’5 Doddie Foundation and the Van Geest Foundation and, incubated by F-Prime Capital and JJDC.

“Whilst neurodegenerative conditions are focal at onset, the pathology eventually spreads throughout the nervous system. We have seen that modifying gene expression can be curative, but achieving widespread distribution is the greatest challenge. We have shown that precision micro dosing to neural networks will deliver broad CNS expression, providing safe and effective treatments,”

said Prof. Christopher Shaw, Co-founder and Chief Scientific and Clinical Advisor of AviadoBio.

“We are directly exploiting insights into the causes of diseases to design therapies that have the potential to cure patients for whom there are no effective treatments. I believe that AviadoBio has the potential to move neurodegeneration from palliation to prevention.”

Shaw added:

“AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery. Precision micro dosing achieves extensive gene expression throughout the nervous system, maximizing the therapeutic potential for patients living with devastating neurological diseases.”

The Company is led by recently appointed Chief Executive Officer and Board member Lisa Deschamps, former Senior Vice President and Chief Business Officer at Novartis Gene Therapies. Lisa brings more than 25 years of biopharma experience including leading the global neuroscience business of Novartis and the worldwide commercialization of its gene therapy for spinal muscular atrophy.

“I am excited about the promise we can bring to people living with neurodegenerative disease, where a significant unmet medical need remains with only symptomatic therapies currently available. Our differentiated approach has the potential to transform the treatment landscape and we have the opportunity with our group of experienced investors, our advisors and founders, to change the lives of people living with these debilitating conditions,”

said Lisa Deschamps, Chief Executive Officer.

“The expansion of our team and investor syndicate, positions AviadoBio to make this mission a reality while advancing the field.”

As part of the Series A financing, Matt McAviney, Partner at NEA and Tim Funnell, Partner at Monograph Capital have been appointed to the Company’s Board of Directors. They are joined by Amber Salzman and Prof. Philip Scheltens as an independent Board Director and a Board Observer for LSP, respectively. Amber has been a pioneering entrepreneur in leading companies and not-for-profit organizations focused on gene therapies and rare diseases for more than 20 years, while Prof. Sheltens is a world-renowned dementia scientist and thought leader.

“Traditionally, there have been very few options to combat the devastating course of neurodegenerative disorders,”

said Matt McAviney, Partner, NEA and AviadoBio Board member.

“AviadoBio’s gene therapy platform introduces a revolutionary approach, supported by world-renowned neuroscience expertise and strong preclinical data. The Company is poised to make a meaningful impact, and we are thrilled to partner with the team to introduce a new portfolio of treatments to patients suffering from a range of debilitating diseases.”

ABOUT AVIADOBIO

At AviadoBio our mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute, co-founded by Prof. Christopher Shaw, Dr. Youn Bok Lee and Dr. Do Young Lee, with support from leading life science investors and Dr. Jonathan Jones and Dr. Graeme Fielder, as the Company’s founding management team. AviadoBio is building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products.

For more information, please visit www.aviadobio.com and follow us at Twitter @AviadoBio and LinkedIn AviadoBio.
LEARN MORE ABOUT US

CONTACT
For media enquiries:

Consilium Strategic Communications
Chris Gardner, Angela Gray, Isabelle Abdou
+44 (0) 20 3709 5700
AviadoBio@consilium-comms.com