Kristofer Mussar on VectorBuilder’s ethical imperative in gene delivery


As genetic technologies unlock new possibilities, companies like VectorBuilder aim to navigate progress responsibly, guided by a strong moral compass. “Ethics to me is the one thing that really is so, so important,” said Kristofer Mussar, managing director of VectorBuilder GmbH. “I have such a high ethical threshold I make decisions that are sometimes not the best business decisions, but they are the right ethical decisions.”

VectorBuilder offers an array of products and services, including vector design, vector cloning,  plasmid DNA preparation, AAV capsid evolution, mRNA gene delivery systems, and CDMO services. Its international customer base includes thousands of labs and biotech/pharma companies.

In business practices, especially in a field such as gene delivery, ethics are tantamount. “If you have very strong ethics, that means you have trust,” he explained. “If everybo…

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Unicorn gene-delivery firm VectorBuilder eyes future breakthroughs

This image from VectorBuilder showcases a comparative study of luciferase expression in various mouse organs, highlighting the targeted gene delivery capabilities of Anti-CD31 conjugated LNP-mRNA. Controls include IgG2a-conjugated FLuc LNP-mRNA and naked FLuc mRNA.

Genetic tools are growing more powerful by the day and hold immense medical promise. Kristofer Mussar, managing director of VectorBuilder GmbH who holds a Ph.D. in molecular genetics and epigenetics, noted that in the wake of the pandemic, genetic research requires increased awareness and ethical stewardship to responsibly tap into its power. While the current financial climate poses challenges, “the future is very bright in the cloning world and gene delivery,” Mussar noted. “At VectorBuilder, we’re all about innovation and having our finger on the pulse. We’re always focusing on the next thing.”

Earlier this y…

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FTC cracks down on 100-plus ‘improper’ drug patents in comprehensive enforcement push

Under the leadership of Lina Khan, the Federal Trade Commission (FTC) has stepped up its enforcement actions, complicating its clearances of mergers, including Amgen’s acquisition of Horizon Therapeutics, which concluded in early October. Most recently, FTC has challenged what it deems are “improper” patent listings. At the receiving end of the FTC drug patent crackdown are Big Pharma companies such as AbbVie, AstraZeneca, and Boehringer Ingelheim. 

FTC drug patent crackdown targets 100+ patent listings

More than 100 patent listings are improper in the eyes of FTC because they do not meet the relevant statutory and regulatory requirements for inclusion in the FDA’s Orange Book, a publication of “Approved Drug Products with Therapeutic Equivalence Evaluations.” That is, FTC contends that the patents in question are either inaccurately listed or are not relevant to the approved drug products they are thought to cover. The agency notes a variety of reasons fo…

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Biden names 31 tech hubs: Here are 10 relevant to pharma and biotech

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Traditionally, the tech and biotech sectors in the U.S. have been concentrated in a handful of regions — most notably in areas such as Boston, Seattle, Silicon Valley and Southern California. But the Biden administration aims to distribute innovation more evenly through the U.S. To that end, the administration has designated 31 tech hubs across the U.S. to help drive innovation and job creation in a variety of industries.

These tech hubs are part of a competitive program involving $500 million in federal funding, with each hub eligible to apply for $50–$75 million to drive innovation in their respective focus areas. Here, we highlight 10 hubs that are especially relevant to the pharma and biotech sectors, describing their unique focus areas.

Three of the regions were featured in an earlier Drug Discovery & Development roundup on emerging biotech hubs: Baltimore, Maryland; Mad…

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How the latest AI executive order might impact drug development in the U.S.

Image courtesy of Wikipedia

The White House has released an executive order that contains what it hails as “the most sweeping actions ever taken to protect Americans from the potential risks of AI systems.” Relevant to drug development, a fact sheet on the order describes its aim to help further “the responsible use of AI in healthcare and the development of affordable and life-saving drugs.” The order also is designed to protect against the risks of using AI to synthesize new chemicals and biological materials, and will require government agencies funding life science projects to follow these standards as a prerequisite.

Other provisions of the order that could affect drug developers include its potential to affect AI-assisted clinical trials. The order could require drug developers to ensure that their algorithms do not exacerbate discrimination in clinical trials or patient interactions. Drug …

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Nobel-connected startup Shasqi deepens J&J partnership on CAPAC platform

San Francisco-based oncology startup, Shasqi, announced an expansion of its research collaboration with Johnson & Johnson Enterprise Innovation. The partnership centers on Shasqi‘s CAPAC platform, which is an abbreviation for Click-Activated Protodrugs Against Cancer. The platform separates tumor-targeting from the actual drug payload with the aim of maximizing potency while minimizing toxic side effects. 

Shasqi’s CAPAC platform taps click chemistry for tumor targeting

CAPAC makes use of a two-part system: a tumor-targeting agent and a cancer therapeutic. When the protodrug locates the tumor’s targeting agent, click chemistry activates it. The technique joins two discrete molecular components much like a seat belt buckle. More precisely, the system makes use of a chemical reaction to precisely activate the cancer drug at the tumor location. 

The origins of Shasqi trace back to the research of its founder, Dr. José M. Mejía Oneto, who desc…

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Ginkgo Bioworks and Google Cloud forge five-year AI and biology partnership

Ginkgo Bioworks’ pioneering capabilities in harnessing vast biological data. [Image courtesy of Ginkgo Bioworks]

Founded in 2008, Ginkgo Bioworks’ stock jumped almost 25% on August 29, hitting $2.22, after unveiling a five-year partnership with Google Cloud. The partnership centers around the development of novel AI tools for biology and biosecurity. In particular, Ginkgo hopes to further its mission to make biology easier to engineer in the AI era.

Opting to make Google Cloud its primary cloud services provider, Ginkgo plans to develop new large language models for biological engineering applications based on Google’s Vertex AI platform. Debuting in 2021 as a framework for streamlining the machine learning lifecycle, Vertex AI has since evolved to incorporate more generative AI capabilities.

Further solidifying the partnership, Google Cloud will also help fund Ginkgo’s development of foundation mod…

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Cellares teams up with Bristol Myers Squibb to explore automated CAR-T cell therapy manufacturing

Less than a week after announcing that it has secured $255 million in Series C funding, South San Francisco-based startup Cellares has revealed that Bristol Myers Squibb has joined its Technology Adoption Partnership (TAP) program. To date, the company has raised more than $355 million in total financing.

As part of the TAP program, Bristol Myers Squibb plans on conducting a proof-of-concept transfer of a CAR-T cell therapy process onto the Cell Shuttle, which the company has called a “factory in a box.”

Cellares plans to use the funding to launch what it dubs the world’s first commercial-scale Integrated Development and Manufacturing Organization (IDMO) smart factory. Koch Disruptive Technologies led the investment round, which included participation from Bristol Myers Squibb, DFJ Growth, Willett Advisors, Eclipse, Decheng Capital and 8VC.

When asked how Cellares managed to receive a large funding round in a difficult financial climate, Gerlinghausen sa…

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Regeneron agrees to spend up to $213M to acquire Decibel Therapeutics 

To date, 2023 is shaping up to be a relatively brisk year for M&A deals. A recent case in point is Regeneron’s acquisition of Decibel Therapeutics. The centerpiece of the deal is DB-OTO, a gene therapy intended for patients with otoferlin-related hearing loss. The drug is in its first clinical trial. Regeneron agreed to pay $4.00 per share for Decibel stock with the potential to pay up to $3.50 per share in cash if Decibel hits agreed-upon regulatory milestones for DB-OTO in a specified time frame, bringing the total value of the deal to roughly $213 million.

Last year, Decibel touted DB-OTO’s ability to support otoferlin expression for several weeks before it plateaued. The company said the data for the drug were in line with earlier preclinical research in which mice achieved a functional recovery.

Involved in transmission of sound signals in the inner ear, the protein otoferlin is essential for hearing. Deficiency or mutations in the gene that produces o…

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Aiming to tame psychedelics’ wild side in pursuit of FDA approval

Doors of perception: Psychedelic renaissance or Pandora’s box?

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In one sense, psychedelics have always been divisive in mainstream Western culture. During their heyday in the 1960s, proponents lauded psychedelics’ virtues for psychological healing and exploration. But troubling reports also emerged — stories of bad trips, psychological breaks and mostly apocryphal yet sensationalized reports of individuals leaping from buildings while under the influence. This darker side, combined with Nixon’s targeting of drugs popular with hippies, cast a pall over psychedelic research for decades, making the prospect of FDA approval seem like a practical impossibility.

Psychdelic resurgence and pursuit of FDA approval

But now, thanks to loosening restrictions, psychedelics are as hip as they have been in decades, and are a popular research subject in many academic circles. In June, FDA released gui…

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Take a global tour of 19 major biotech hubs with our interactive map

The biotech sector clocked in at $723 billion last year despite headwinds such as slow launches, long-term drug pricing shifts and changing consumer behavior. Recent biotech breakthroughs include the development of an Ebola vaccine, progress in genetic testing and drug treatments and advances in artificial tissue growth. This article explores several leading biotech centers across the world and examines their distinct attributes and contributions.

1. Boston & Cambridge, Massachusetts

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Innovation Landscape: A powerhouse in the biotech and education sectors, with over 1,000 biotech companies, ranging from small startups to billion-dollar firms. High patent activity reflects a vibrant innovation ecosystem. Academic Pedigree: Home to top-tier universities such as MIT and Harvard, which feed the regional innovation network with a constant flow of scientific talent and research. Investmen…
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