Lisa Deschamps, AviadoBio CEO
Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.
‘So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,” he told Endpoints News.
In 2019, he launched AviadoBio along with molecular neurobiologist Youn Bok Lee and vector biologist Do Young Lee to pursue a new take on gene therapy for neuro disorders. And on Thursday, they unwrapped an $80 million Series A round led to bring their lead candidate for frontotemporal dementia (FTD) into the clinic. ‘I was an instant believer,’ said Lisa Deschamps, who left her role as CBO of Novartis Gene Therapies to take the helm at AviadoBio in October.
The team is targeting diseases that have an aggressive early progression, Shaw said. While most of their competitors are putting vectors into the cerebrospinal fluid — a liquid found within the tissue that surrounds the brain and spinal cord — AviadoBio is looking to deliver the vectors right to the brain.
‘We get fantastic expression by doing that,’ Shaw said. ‘It also allows us to microdose — we’re giving about a thousandth of the dose of our competitors.’
Their lead candidate, AVB-PGRN, and an intrathalamically dosed therapy designed to deliver a functional copy of the progranulin (GRN) gene. FTD is caused by a deficiency of progranulin, which plays a role in lysosomal function, or the protein recycling part of the cell.
‘Neurons are incredibly dependent on having good recycling because the neurons you die with are the neurons you’re born with,’ Shaw said. ‘So you know, they can’t be adolescents. They can’t have a messy room. They’ve got to have a really, really crisp, tidy space … So what we’re trying to do is just return back to physiological levels this really important recycling protein.’
AviadoBio will compete with a handful of players working on potential treatments for FTD, including GSK-partnered Alector, which unveiled data this summer showing its antibody AL001 brought progranulin levels back to near-normal levels after a half-a-year. And Expansion Therapeutics unveiled an $80 million Series B round just a couple months ago to bring its small molecule RNA platform one step closer to the clinic – including a candidate for FTD.
Deschamps said AviadoBio’s candidate should enter the clinic by the end of next year, while another preclinical candidate for ALS is a bit further behind. The Series A round was led by New Enterprise Associates and Monograph Capital. A handful of big-name backers pitched into a $16.5 million seed round last year, including Advent Life Sciences, Dementia Discovery Fund (DDF), F-Prime Capital, Johnson & Johnson Innovation (JJDC), and LifeArc.
Despite Big Pharma’s retreat from neuro a few years ago, the industry seems to be warming back up to the field, Deschamps said — and new companies are emerging with gene therapies to take a shot at difficult-to-treat conditions. This past summer, for example, Alcyone Therapeutics emerged from stealth with $23 million and 12 gene therapies in tow, with a rare neurodevelopmental condition called Rett syndrome up first.
‘I was leading the global neuroscience business at Novartis … one of the things we had talked about years ago was how many Big Pharma companies were exiting the space. It is quite the opposite these days,’ Deschamps said. ‘We see certainly a resurgence across big and small companies, you know, coming back into the neuroscience space.’
While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.
Merck’s new antiviral molnupiravir (Quality Stock Arts / Shutterstock)
After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.
‘I think it is super worrisome,’ Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, ‘This is not going to be good.’
Unlock this story instantly and join 124,400+ biopharma pros reading Endpoints daily — and it’s free.
Fujifilm Diosynth Biotechnologies is in the middle of a monumental point in the company’s 10-year history, and the CDMO is about to grow even more, as it sets out to be the ‘beating heart’ of the UK’s North East Life Sciences ecosystem.
A site in Billingham, Teeside, UK will receive a $453.72 million investment package from the manufacturer to double the existing footprint and create the largest multi-modal biopharmaceutical manufacturing site in the UK, bringing another 350 jobs to the region by late 2023.
Unlock this story instantly and join 124,400+ biopharma pros reading Endpoints daily — and it’s free.
Radek Spisek, Sotio CEO (Cellestia)
In the rather insular world of biotech, most innovation inevitably comes from a cluster of R&D hubs — Cambridge, San Francisco, etc. But sometimes success stories sprout from rocky soil, which is most certainly the case with Prague-based Sotio Biotech and its suddenly jam-packed pipeline of cancer drugs.
After years in quiet development, Sotio now has $315 million in new funds to play with from parent company PPF Group, an investment group founded in the Czech Republic, as the biotech looks to advance its growing pipeline through early- and mid-stage trials.
Unlock this story instantly and join 124,400+ biopharma pros reading Endpoints daily — and it’s free.
Samsung Biologics has entered an agreement with South Korean biotech GreenLight BioSciences to manufacture its mRNA Covid-19 vaccine at commercial scale, the two companies announced.
Samsung Biologics, one of the fastest growing manufacturers in the world right now, will use its vaccine manufacturing expertise to help patients in lower-income countries, CEO John Rim said in a press release. This will help expand their capabilities from drug substance to aseptic fill-finish and all the way to commercial release from one site.
Vas Narasimhan, Novartis CEO (Thibault Camus/Pool via AP Images)
Thursday marks Novartis’ annual R&D day, and with it comes CEO Vas Narasimhan’s attempt to spotlight the company’s pipeline strategy and emerging stars.
The biggest question entering Thursday’s presentation dealt with how the big biopharma will make up revenues from upcoming generic competition — Novartis says within the next five years, generics will eat away roughly $9 billion in sales. To offset this, Narasimhan outlined a strategy for 4% growth or higher until 2026, focusing on six key medicines he believes will see multibillion dollar profits during this time.
The US Court of Appeals for the Federal Circuit on Wednesday rejected Moderna’s attempt to overturn key patents related to the delivery vehicle for its Covid-19 vaccine after the biotech sought to preempt a potentially risky infringement lawsuit.
For years, Moderna has been battling a tiny Pennsylvania biotech known as Arbutus over patents for a technology required to deliver its mRNA drugs and vaccines, known as lipid nanoparticles or LNP. Moderna is concerned there’s a substantial risk that Arbutus will assert the ‘069 patent in an infringement suit targeting Moderna’s Covid-19 vaccine, particularly as Arbutus has boasted of its patent protection and refused to grant a covenant not to sue Moderna.
Unlock this story instantly and join 124,400+ biopharma pros reading Endpoints daily — and it’s free.
On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.
Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.
GenScript, Suzhou Abogen, and Walvax Biotechnology have announced the three companies will be collaborating on a Covid-19 mRNA vaccine project dubbed ABO-O28M.
WalVax will submit a BLA for the project, and GenScript will provide exclusive manufacturing services, according to Asia One.
The project stems form an agreement between Abogen, the Academy of Military Medical Sciences (AMMS) of the PLA Academy of Military Science and Walvax, which gained clinical trial approval in June 2020. It was one of the first vaccine projects approved by China’s government, and GenScript used its plasmid GMP manufacturing to accelerate into clinical trials.
https://endpts.com/ex-novartis-business-head-hops-over-to-a-gene-therapy-startup-and-shes-reeled-in-80m-for-a-dash-to-the-clinic/
