Cambridge, UK, 1st April 2026 - Broken String Biosciences, today announced the submission of a Master File (MAF) to the U.S. Food and Drug Administration (FDA) for its �����ٱ��䷡-�����® platform, marking a significant step in supporting regulatory adoption of genome-wide DNA break analysis in gene editing programs. 

The filing reflects increasing regulatory focus on comprehensive, cell-based assessment of on- and off-target effects in investigational new drug (IND) submissions, which �����ٱ��䷡-�����® is designed to address. As expectations evolve, developers are under growing pressure to generate robust, genome-wide data early in development to support safety and decision-making. 

The �����ٱ��䷡-�����® master file provides FDA reviewers with direct access to detailed technical, validation and performance data describing the platform, without requiring sponsors to include proprietary information in their own regulatory submissions. 

This enables gene editing teams to reference the platform through a Letter of Authorization (LOA), streamlining IND preparation while maintaining full protection of intellectual property. 

�����ٱ��䷡-�����® is a cell-based, PCR-free platform designed to directly capture and map DNA breaks across the genome. The platform delivers quantitative, unbiased insight into both on- and off-target activity within days, supporting earlier, more confident decisions and reducing risk as programs progress toward the clinic. 

The master file includes comprehensive documentation covering platform design, analytical performance, software validation, and manufacturing and quality systems, providing a structured framework for regulatory review.  

Terry Pizzie, CEO, ���Ҵ�ý, said: “Regulatory expectations for genome-wide off-target analysis are evolving quickly, and teams need approaches that can stand up to that scrutiny. Too many programs are still relying on methods that are indirect, slow or difficult to scale, which creates risk later in development. 

By filing the �����ٱ��䷡-�����® master file, we are making it significantly easier for our partners to incorporate high-quality, genome-wide break data into their regulatory strategy, without adding complexity to their submissions. It’s about enabling better decisions, earlier, and supporting programs as they move toward IND.” 

The master file has been formally submitted to the FDA and acknowledged, with an assigned reference number. It will be reviewed by the agency when referenced within a sponsor’s IND submission. 

As a living regulatory document, the master file will continue to be updated with additional data and validation, ensuring that sponsors referencing �����ٱ��䷡-�����® benefit from the most current version during regulatory review. 

To learn more about ���Ҵ�ý and how �����ٱ��䷡-�����® supports regulatory-ready genome-wide DNA break analysis, please visit: 

Cambridge, UK, 31 March 2026 – ���Ҵ�ý, a biotechnology company enabling gene editing teams to accelerate development of safer, more effective genetic medicines, today introduced new branding and comprehensive website as part of its strategic repositioning. At a time where the gene editing industry is facing increasing regulatory demands for genome-wide off-target data, the update has been made to reflect the ���Ҵ�ý’s transition from a specialist technology provider to a commercially focused partner, supporting early discovery programs through to investigational new drug (IND)-enabling studies.

���Ҵ�ý’ new identity signifies the next stage in its ongoing commercial development, following the appointment of life science leader, Terry Pizzie, as CEO in November 2025¹. The update has been introduced to better align how the ���Ҵ�ý presents itself with the capabilities it has established in the �����ٱ��䷡-�����® platform – a complete in-house solution for genome-wide detection and quantification of DNA breaks. With the redesigned website, researchers can easily access a comprehensive resource library and gain a clear understanding of how the platform can be applied across gene editing workflows to maximize value in development pipelines.

Off-target DNA breaks are a key safety concern in developing gene-edited cell and gene therapies. Regulatory expectations are shifting rapidly, with genome-wide, cell-based off-target assessment becoming a requirement for IND submissions. Existing approaches are often indirect, slow and difficult to scale, limiting their usefulness in decision-making and regulatory submissions.

The �����ٱ��䷡-�����® technology was developed to address these limitations. The platform is scalable, cell-based and PCR-free, directly capturing and mapping individual induced and endogenous breaks at the point of formation within cells. Designed for use in-house, it provides precise, quantitative and unbiased insights into on- and off-target activity across the genome within days, enabling earlier, more confident decisions before programs reach critical inflection points. The platform also delivers deeper understanding of editing kinetics, nuclease behaviors and cell repair pathways, enabling researchers to streamline preclinical decision-making and de-risk therapeutic development.

Terry Pizzie, CEO, ���Ҵ�ý, said: “Understanding DNA break activity in real cellular contexts is now a fundamental expectation for therapeutic programs. Yet, too many programs are still relying on approaches that are indirect, slow, or difficult to scale and that creates risk later in development. Our technology allows researchers to generate high-quality, genome-wide safety data in their own labs, early enough to influence decisions. This rebrand and website launch reflects that mission, providing customers with greater clarity about the role we play in helping teams move forward with confidence.”

To learn more about ���Ҵ�ý and the �����ٱ��䷡-�����® platform, please visit: www.brokenstringbio.com

1) Press Release (17th November, 2025): ���Ҵ�ý Announces New Leadership to Drive Commercialization of INDUCE-seq Platform for Gene Editing On- and Off-target Characterization

Media Enquiries

���Ҵ�ý
Hannah Ingram
Email: hannahingram@brokenstringbio.com

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]]>���Ҵ�ý Rebrands to Reflect Strategic Evolution as Demand Grows for Unbiased Genome-wide Off-target AnalysisPress ReleaseJamie HarmesMon, 17 Nov 2025 16:13:10 +0000/news/broken-string-biosciences-announces-new-leadership-to-drive-commercialization-of-induce-seq-platform-for-gene-editing-on-and-off-target-characterization69429e7198b87e513d8a40ea:69440d741a3491229c801e0a:69440d741a3491229c801e0b

CAMBRIDGE, UK – November 17th, 2025 – ���Ҵ�ý (“Broken String”), a leader in advancing gene editing safety, today announced the appointment of new leadership to drive the commercialization of its �����ٱ��䷡-�����® platform, now available to cell and gene therapy developers worldwide.

To drive the company’s commercial expansion, Terry Pizzie has been appointed as CEO, bringing extensive experience from leading and scaling global life science businesses. Mr. Pizzie has previously held leadership positions, including CEO of Horizon Discovery and Astrea Bioseparations, where he guided both companies through successful growth and exits, as well as senior roles at Pacific Biosciences, GE Healthcare, Applied Biosystems, and Genetix. His appointment marks a significant milestone as Broken String transitions from a pioneering technology developer to a fully commercial organization.

The company’s next stage of growth is focused on scaling access to �����ٱ��䷡-�����®, a proprietary platform that directly maps DNA double-strand breaks to measure and quantify both on- and off-target editing events from any gene editing therapeutic in any cell type. �����ٱ��䷡-�����® is a universal, unbiased solution for mapping genome integrity and was developed to provide a faster and more reliable way to quantify such events, which is critical in evaluating the predicted impact of any gene editing therapy.

Co-founder and former CEO, Felix Dobbs, will continue to lead company operations and technology development as Chief Operations Officer. Gabe Longoria joins as Chief Commercial Officer, his significant track record of commercial leadership in Cell & Gene Therapy tools adding further depth to the company’s commercial expertise. Co-founder Professor Simon Reed continues in his role as Chief Scientific Officer.

“Terry’s proven track record in building commercially successful life science businesses makes him the ideal leader to guide Broken String through its next phase,” said Felix Dobbs. “Under his leadership, and with Gabe’s commercial acumen, we are now well positioned to make �����ٱ��䷡-�����® the gold standard for gene editing off-target assessment, a crucial step in the delivery of safe gene editing therapies to patients.”

“Gene editing technologies such as CRISPR and base editing are already transforming what’s possible in medicine,” said Terry Pizzie. “�����ٱ��䷡-�����® gives developers a powerful new way to rapidly and confidently evaluate both the efficacy of on-target editing and the potential impact of off-target effects, helping them design more precise, safer therapies and to advance promising therapies much faster. This is an exciting moment for the company and the industry, and I’m thrilled to lead Broken String as we bring this technology to more customers globally.”

Recent data presented at the Cell & Gene Meeting on the Mesa 2025, including work with the Innovative Genomics Institute at UC Berkeley, showcased how �����ٱ��䷡-�����® accelerates early-stage gene editing therapeutic development by cutting candidate screening timelines from months to days.

About ���Ҵ�ý

���Ҵ�ý is advancing more precise, safe, and effective cell and gene therapies through its cutting-edge technology platforms. The company’s core platform, �����ٱ��䷡-�����®, was developed as the new gold standard for precise mapping of DNA breaks to measure and quantify on- and off-target genetic edits, crucial for ensuring the efficacy and safety of advanced therapies from discovery through commercialization.

About �����ٱ��䷡-�����®

Broken String Bioscience’s �����ٱ��䷡-�����® enables direct, cell-based measurements of on-target and off-target edits directly from a clinically relevant biological system. This ensures therapy developers can characterize safety and efficacy as early as discovery, before committing substantial budgets and time to preclinical or clinical trials. The technology also allows for off-target assessments tailored to a patient’s specific genotype, enabling developers to bring safety evaluations closer to the patient. This capability supports faster, more targeted treatments, helping to accelerate progress in life-saving therapies.

Media Contact

brokenstringbio@consortpartners.com

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]]>���Ҵ�ý Announces New Leadership to Drive Commercialization of �����ٱ��䷡-�����® Platform for Gene Editing On- and Off-target Characterization���Ҵ�ý NewsJamie HarmesTue, 05 Aug 2025 15:12:55 +0000/news/broken-string-biosciences-renews-commitment-to-nist-genome-editing-consortium-bolstering-efforts-to-standardize-gene-editing-safety69429e7198b87e513d8a40ea:69440d741a3491229c801e0a:69440d741a3491229c801e11BOSTON & CAMBRIDGE, England–() –  (“Broken String”), a leader in advancing gene editing safety, today announced its formal re-engagement as a member of the National Institute of Standards and Technology (NIST) Genome Editing Consortium. Broken String plays a key role in developing the gold-standard of safety testing for gene-editing therapies, based on its �����ٱ��䷡-�����® platform. INDUCE-seq is commercially deployed today to address a critical challenge in the safe development of gene therapies: identifying unintended edits that may activate oncogenes or disable tumor suppressors.

Broken String’s participation in the NIST Genome Editing Consortium is part of the company’s efforts to shape industry-wide safety standards for emerging genome editing technologies, including CRISPR, base editing, and prime editing. The company continues to partner with the consortia and other leaders in the field to publish findings and engage with regulators to promote greater understanding and adoption of its breakthrough technology. One such effort is the cross-sector Health and Environmental Sciences Institute Cell and Gene Therapy – Tracking, Circulation, & Safety (HESI CT-TRACS) Committee, where 30 like-minded partner companies – including major biopharma partners – are helping advance breakthrough technologies that improve human health.

“Gene editing holds enormous promise, but without standardized tools to assess both on- and off-target edits, developers will continue to face challenges in demonstrating safety to regulators and the public,” said Felix Dobbs, CEO and co-founder of Broken String. “Our ongoing collaboration with NIST and leading industry partners such as AstraZeneca and Novartis underscores the urgency of our work. We believe that implementing INDUCE-seq early in the development process can significantly reduce the risk of costly regulatory holds and adverse effects, protecting both patient safety and business investment. This is how we help realize the full potential of life-changing gene therapies.”

This renewed collaboration underscores Broken String’s commitment to advancing the safety and standardization of gene-editing therapies through its innovative INDUCE-seq platform. INDUCE-seq is a technology intended to help standardize the gene therapy industry. The NIST Genome Editing Consortium is dedicated to generating crucial data that Broken String believes will highlight the superior features of INDUCE-seq, a PCR-free platform that enables unbiased, high-resolution detection of double-strand DNA breaks (DSBs). This includes its reproducibility, robustness, and sensitivity, surpassing existing approaches in off-target detection.

For more information on the NIST Genome Editing Consortium, visit .

Powered by gene editing advances like CRISPR and base editing, cell and gene therapy (CGT) is delivering on the promise of genomic medicine. First-generation CAR T-cell therapies, for example, have been functional cures for many patients with otherwise untreatable hematologic cancers. Newer gene editing tools continue to improve CGT safety, efficacy, and specificity. For more complex diseases, however, the next wave of therapies may require multiple, complex edits within a single cell.

The success of these therapies hinges on the accuracy of on- and off-target edits. Notably, concerns over off-target effects have been cited in several FDA clinical holds for CGTs.1 Until recently, identifying and quantifying genome breakage patterns relied on methods that were slow, expensive, or prone to PCR bias.

The collective measurement of all DNA breaks in a genome is known as the “breakome.” Emerging next-generation sequencing (NGS) technologies now allow us to measure the breakome, with implications far beyond CGT safety.

Depiction of breakome profiling to find on- and off-target CRISPR edits. [Broken String]

The breakome is a new layer in the multiomics landscape—alongside the genome, transcriptome, proteome, and interactome. While it hasn’t traditionally been included among these other omics, we consider it a significant new dimension.

Exploring the breakome

DNA breaks result from both external and internal sources—environmental exposures and biological aging, for example. Among these, double-stranded breaks (DSBs) are the most severe, causing genome instability and mutagenesis.

DSBs have been linked to diseases ranging from neurodegenerative disorders to cancer. When erroneously repaired, they can drive cancer progression. DNA instability enables cancer cells to clonally expand, and cancer therapies often exploit DNA damage response (DDR) pathways to selectively kill these unstable cells.

We know now that genome breaks do not occur randomly. Specific regions of the genome are more prone to breakage, and patterns vary by tissue, cell type, genetic background, and environmental exposure. By studying these patterns, we can uncover mechanisms of disease as well as identify unintended consequences of therapeutic genome editing, which often begins with a DSB.

Genotoxicity to breakome mapping

The INDUCE-seq platform grew from our work at Cardiff University, initially focused on measuring CRISPR-induced genotoxicity and DNA damage. In genotoxicology, any new compound must be tested for its ability to cause DNA breaks, as these are direct drivers of cancer. Genotoxicity data is a prerequisite for drugs before they enter Phase I clinical trials, forming the basis of a drug’s risk-benefit profile and playing a large role in determining whether it is safe for human use.

Despite advances in drug development, genetic toxicology has lagged in adopting novel technologies. Originating after World War II from nuclear radiation research, its standard assays remain largely unchanged: simple, cell-based, and low-resolution. For example, the comet assay detects DNA fragmentation via smeared bands in a gel—inadequate for this digital age of data-driven medicine.

Some methods, like the micronucleus test for DNA breaks in vivo, are also time- and cost-intensive. As a result, chronic toxicity studies can last six months to two years, requiring techniques like high-dose exposure in rodents before evaluation of DNA breaks can be made.

Modernizing break detection with NGS

Multiple academic groups have looked to modernize our ability to study the breakome. Mapping DSBs at single-base resolution using NGS has now been achieved through a variety of PCR-based sequencing techniques. One of the first was called Breaks Labeling, Enrichment on Streptavidin, and Sequencing (BLESS), which labeled DSBs in situ before genome-wide mapping. Ground-breaking in its time, the method’s sensitivity was low, and it initially required several million cells to perform. Subsequent iterations were able to improve on one issue or the other, but ultimately, all PCR-based methods of its type are subject to amplification bias.2

These challenges led us to develop INDUCE-seq, which is built on a PCR-free NGS flow cell enrichment concept. Because it does not rely on PCR, it avoids that bias, ensuring each sequencing read derives from a single, labeled DSB. In a Nature Communications publication, we demonstrated that the platform can detect DSBs induced by CRISPR-Cas9 genome editing, as well as its potential for measuring off-target edits.3 The platform can also be used to measure aggregate levels of genome stability, which has applications in multiple clinically relevant settings.

Unlocking the secrets of the breakome

Despite recognition of the many biological implications of DNA breaks, no one has yet provided a solution that enables a complete, breakome-wide view. INDUCE-seq was developed for this purpose—and soon proved useful beyond genotoxicity testing. Its clean data and fast turnaround have opened new avenues, many of which are still being discovered.

One ready application is cancer diagnostics. Revealing certain patterns of DNA breaks could be used to diagnose disease, identify a subtype, or even determine how a patient is responding to a treatment.

Drug development is another exciting application area. Several companies are advancing DDR-targeted therapies, including PARP inhibitors, some of which have already been approved. These selectively kill cancer cells by exploiting high levels of unrepaired DNA breaks in the cancer cells that are repaired in healthy cells. Having a detailed genome-wide view of break induction by emerging DDR modalities could enable the development of new classes of these drugs with novel mechanisms of action.

Breakomics may also guide the use of PPAR agonists, which induce apoptosis via DDR modulation. It could inform radiotherapy and chemotherapy strategies that deliberately cause breaks to kill tumors.

Gene editing therapies: A key use case

Among the most exciting near-term applications is gene editing therapy. Technologies like CRISPR and base editing rely on DSBs—directly or indirectly—as the first step of editing. Delivering edits safely means developers must demonstrate a low risk of off-target activity to regulators.

Currently, companies often create in-house assays and bioinformatics tools to evaluate off-target edits. These workflows can take weeks or months. INDUCE-seq shortens that timeline to just days, offering high-resolution break mapping and a full breakome profile.

This speed allows iterative use not just in late-stage testing, but throughout drug discovery. In addition to safety, breakome data provide insight into on-target edits and therapeutic function. Future versions of INDUCE-seq will run in developers’ labs, increasing scalability and efficiency.

Looking ahead

We believe that INDUCE-seq offers a solution for interrogating the breakome, particularly for gene editing. Our collaborators are also looking at new ways of leveraging the platform. Some groups, for example, are interested in identifying break hotspots in the genome that correlate to specific diseases, including neurodegenerative disorders such as amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease).

Another interesting approach being used is AI: we have already developed algorithms that can identify natural vs. induced breakage patterns from the remarkably clean data produced by INDUCE-seq. This enables us to identify off-target editing based purely on

real-world datasets. The next step is to see if we can train an AI to predict the mutational outcomes of breaks entirely in silico.

As with many advances in genomics technology, we do not yet know all the applications that INDUCE-seq can be used for. But we do see the potential for novel breakome-derived insights to change how we understand cellular function and disease development, while helping deliver a promised generation of therapeutics unlocked by the power of genomics.

Learn More about �����ٱ��䷡-�����®

References

1. Lu S, Wang G, Albino Bacolla, Zhao J, Spitser S, Vasquez K. . Cell Reports. 2015;10(10):1674-1680.

2. Pfeifer GP, Jin SG. Methods and applications of genome-wide profiling of DNA damage and rare mutations. Nature Reviews Genetics. 2024; 25, 846–863. doi:https://doi.org/10.1038/s41576-024-00748-43.

3. Dobbs FM, van Eijk P, Fellows MD, Loiacono L, Nitsch R, Reed SH. . Nature Communications. 2022; 13, 3989.

BOSTON & CHICAGO & CAMBRIDGE, England–()–���Ҵ�ý, a leader in advancing gene editing safety, and BioLizard, an expert in scientific data analytics and AI, today announced the development of SafeGuide, an AI tool for selecting safer, better guide RNAs (gRNA), the gene editing element that tells tools like CRISPR where to repair DNA. With SafeGuide, developers of next-generation gene-edited therapies will be able to quickly find the ideal gRNA with less reliance on time-consuming and expensive wet lab testing, helping to replace current trial-and-error approaches that often take one to two years and result in drug costs of $4 million and more per patient.

The project benefits from a $935,000 grant through the highly competitive Eurostars-3 program. Eurostars is part of the European Partnership on Innovative SMEs, a program co-funded by 37 participating countries and the EU’s , designed to drive transnational innovation collaborations. As a strategic partner, Broken String and BioLizard worked closely with Moore Grants & Incentives (MGI) to help shape the project in line with their ambitions, contributing not only to the funding success but also to the content and vision that made this proposal stand out.

The development of SafeGuide combines the strengths of the two companies: Broken String will contribute data and expertise from its �����ٱ��䷡-�����® platform. �����ٱ��䷡-�����® can pinpoint where on- and off-target edits occur both naturally and through gene editing methods like CRISPR with high sensitivity, offering crucial insights to train SafeGuide’s AI models to distinguish safe versus high-risk gRNA designs. BioLizard will develop predictive algorithms that integrate Broken String’s genomic break data with other sequence and structural features to recommend gRNAs with high on-target efficacy and low off-target risk.

Felix Dobbs, COO at ���Ҵ�ý added: “This collaboration with BioLizard will set a new standard for safety in genome editing. By using AI to predict and distinguish natural and unnatural DNA break patterns, human intervention is significantly reduced, while safety increases.”

�����ٱ��䷡-�����® is already in widespread use by drug developers to both confirm the on-target edits that drive efficacy and identify off-target edits that can become safety issues. In addition to being available as a lab-based service, Broken String recently announced the availability of its  which makes �����ٱ��䷡-�����® available as on-demand service for select gene therapy developers to detect on- and off-target edits at high resolution, in days, in their own labs.

“We are bringing together our bioinformatics and AI expertise with Broken String’s unique breakome identification capability to create SafeGuide,” said Liesbeth Ceelen, CEO at BioLizard. “SafeGuide tool will significantly reduce the need for extensive off-target screening, ultimately accelerating gene editing development and reducing R&D costs.”

About ���Ҵ�ý

���Ҵ�ý is advancing more precise, safe, and effective cell and gene therapies through its �����ٱ��䷡-�����® platform.

�����ٱ��䷡-�����® has become the gold standard for precise identification of DNA breaks before they become safety issues. INDUCE-seq is in widespread use by drug developers to detect on- and off-target genetic edits and is crucial for ensuring the efficacy and safety of advanced therapies.

From discovery through commercialization, �����ٱ��䷡-�����® is available as a lab-based service and also via the on-demand  which enables gene therapy developers to detect breaks at high resolution, in days, in their own labs.

About BioLizard

BioLizard is a leading multi-national bioinformatics, data analytics and data management service and consulting company heading digital transformation in the life sciences industry. Headquartered in Ghent, Belgium, BioLizard is the trusted go-to partner for data strategy and execution with companies across the range from drug discovery to clinical research and diagnostics as well as animal health and food & agriculture. BioLizard accommodates a uniquely qualified team of 50+ experts, “The Lizards”, who bring together their expertise and abilities covering data management, software engineering, bioinformatics, advanced data analytics, and AI. Their joint backgrounds in biology and computer sciences enables them to apply specialist understanding to each client’s data environment, to provide insights and tools that are aligned with the client’s goals and maximize R&D return on investment. With the proprietary Bio|Verse® platform BioLizard takes the next step in providing bespoke software solutions by tailoring its established programs towards specific client needs. For further information, please visit  and follow us on .

A generation ago, writing a piece like this about using artificial intelligence to develop genome-editing medicines would have sounded like a Star Trek plotline. But both fields have matured to the point where they are becoming synergistic, and the concept of AI guiding targeted and curative drug development is not very far off.

Over the last decade,  have applied machine learning, deep learning, and other models for various CRISPR-related activities, mostly in attempts to predict on- and off-target activity. For the latter, accurate off-target predictions would be a powerful tool for safety assessment, given that opacity has already  leading to clinical holds by FDA. Today, gene-editing drug developers most typically spend a lot of time assembling bespoke analytical workflows to characterize any off-target edits made by their therapy and demonstrate safety. And in drug development – particularly clinical development – every additional moment comes with ballooning costs.

Newly commercialized, out-of-the-box solutions for off-target detection can reduce costs and shrink timelines, but they are still utilized later in development, when problems and mistakes are most costly. In the same vein, tools to quickly characterize on-target editing will soon accelerate initial gRNA selection and iteration, meaning developers will have meaningful early insights into therapeutic efficacy as well.

The goal of AI efforts has been to design safe, efficacious therapies before stepping foot in a wet lab, largely removing the trial-and-error nature of gene-editing therapy development today. No models have yet proved suitable, in large part because they have been trained on messy public datasets or even synthetic data.

Garbage out – permanently

The key, then, is cleaner data. Most attempts to characterize the breakome–the collective measurement of all DNA breaks in a genome–have relied on PCR technology, which introduces amplification bias. However, newer tools rely on PCR-free methods like NGS flow cell enrichment, significantly decreasing noise in the dataset.

Researchers are already  of AI to ultraclean data. Ideally, they will find that DNA breakage occurs in patterns, which will lead to models capable of distinguishing gene-editing-induced breaks from natural ones, without human intervention.

The next step will be to train those new  models on ultraclean breakome data that can be used to distinguish safe from high-risk gRNA design. The aim is to integrate breakome data with other sequence and structural features for predictive algorithms that can recommend gRNAs with low off-target risk and high on-target efficacy. 

Lowering drug development costs

The urgency for such approaches is driven by the astronomical cost of gene-editing therapies. We are in a time of technical marvels, like the first successful personalized CRISPR therapy, developed for an infant with a rare CPS1 deficiency in just eight months. (Four of those months were spent characterizing the off-target edits.) At the same time, medicines like those will remain out of reach for most patients, given that gene therapies can cost as much as $4.25 million for a single dose.

Today’s empirical approach, while yielding some groundbreaking therapies, is inherently unsustainable for broad adoption. Sticker prices can’t come down until R&D costs come down. That requires minimizing the years spent in wet lab testing, reducing trial and error, and securing regulatory cooperation toward a platformized approach to gene-editing therapy.

The vision, especially for rare disease treatments, would be for a company to develop the machinery for a gene-editing platform that it can sufficiently demonstrate is safe and effective, such that it can swap in a new gRNA specific for each therapeutic target it pursues. (FDA recently began such platform technology designations with gene therapy vectors.) From there, the AI would guide selection of the gRNA most likely to be safe and efficacious, followed by rapid confirmation on a gold-standard, out-of-the-box breakome analytical tool. The developer could then jump right into clinical testing – without the need to repeat rigorous preclinical safety studies.

If you notice that this envisioned process barely resembles drug development as we know it – that’s the point. AI is no panacea, but it can have a role in turning artisanal, trial-and-error drug development into a rapid, cost-effective, data-driven process. We are closer than most people realize.

This post appears through the MedCity Influencers program. Anyone can publish their perspective on business and innovation in healthcare on MedCity News through MedCity Influencers.

BOSTON, MA and Cambridge, UK, February 6, 2025 – ���Ҵ�ý (“Broken String”), a leader in advancing gene editing safety, today opened its Catalyst Early Access Program (EAP) for developers of gene-edited therapies that allow for the detection of on- and off-target edits at high resolution, in days, in their own labs. The program allows select companies to use Broken String’s �����ٱ��䷡-�����® platform as a first-of-its-kind offering for rapidly detecting on-target and off-target effects that can occur during gene editing. This new on-demand offering enables gene editing therapy developers to assess on- and off-target effects in days, as opposed to months.

The offering can be integrated into early therapeutics development at target validation, reducing risk and removing the need for costly internal workflows that slow development progress. It is cell and nuclease agnostic, enabling direct measurement in clinically relevant samples, addressing regulatory scrutiny around unintended edits.

Felix Dobbs, Ph.D., co-founder and Chief Executive Officer of Broken String said: “The gene editing industry has reached an inflection point, where the momentum required to reach its potential can only be sustained by expanding patient access – which requires standardization, faster drug development, and robust safety characterization. Our Catalyst program will enable faster and broader industry-wide adoption of �����ٱ��䷡-�����® and position this as an essential technology for the next wave of gene-editing based therapeutics.”

For years, companies have been using �����ٱ��䷡-�����® via Broken String’s labs to ensure safety before entering clinical trials. �����ٱ��䷡-�����® solves a fundamental safety challenge that dates back to the origins of gene editing: how to assess on- and off-target effects as they happen. By making the same technology available for developers in their own labs, they retain full control over their experiments while incorporating safety throughout the entire development process.

The industry lacks a gold standard for detecting on- and off-target edits. Current approaches can have PCR-induced bias, and miss low-frequency unintended edits that are potentially dangerous and can have disastrous consequences for clinical programs. �����ٱ��䷡-�����® improves on existing technologies by delivering unbiased genome-wide insights, accurately detecting double-strand DNA breaks from CRISPR and other gene editing technologies. 

Broken String’s  on-premise Catalyst EAP accelerates discovery programs with iterative use to screen guide RNAs based on efficacy, as measured through on-target editing efficiency, as well as also providing quick indications for off-target safety. Enrollment in the Catalyst EAP is limited to a select group of participants.

Cambridge, UK, 23 September 2024: ���Ҵ�ý (“Broken String”), a genomics company enabling development of the next generation of more precise, safe, and effective cell and gene therapies, today announced the appointments of Laurence Reid, PhD, as Chairman, and Brad Crutchfield as Non-Executive Director (NED) of its Board of Directors. 

Bringing extensive commercial and leadership expertise to the Board, Laurence and Brad will play a pivotal role in providing strategic oversight as Broken String prepares for its next phase of commercial growth. Working closely with CEO Felix Dobbs, PhD, they will help advance the company’s �����ٱ��䷡-�����® product offering to address the significant unmet needs of its global customer base within the expanding cell and gene therapy market.

Laurence is a seasoned biotech entrepreneur with a track record of leadership and company-building success within the sector, previously CEO at Decibel Therapeutics (acquired by Regeneron Pharmaceuticals) and Warp Drive Bio (merged with Revolution Medicines). He served as entrepreneur in residence at Third Rock Ventures, focused on novel drug discovery and development opportunities. Prior to this, he held C-level executive positions at Alnylam Pharmaceuticals, Ensemble Discovery and Millennium Pharmaceuticals. Laurence now applies his expertise in an advisory capacity, serving on the boards of a number of biotech companies. He is also a board member at The Possible Zone and an advisor to Life Science Cares and Mount Auburn Hospital.

Brad brings nearly 40 years’ experience in the development and commercialization of life science tools to Broken String’s Board. Most recently, he served as Chief Commercial Officer of 10x Genomics, building the global sales and support organization that today drives over $600 million in business, and working closely with the CEO to support the ���Ҵ�ý through its IPO in 2019. Previously, Brad has held several leadership positions, including as Senior Vice President, Life Sciences at QIAGEN, Vice President and General Manager, EMEA at Illumina, and President of the Life Science Group at Bio-Rad Laboratories. He currently sits on the Boards of Pixelgen AB, Partillion Inc and S2 Genomics, in addition to serving as Senior Advisor for Alamar Biosciences. 

“Laurence and Brad bring distinguished careers and a strong history of driving impact and growth within our industry. It is a privilege to welcome them to the Broken String team,” said Felix Dobbs, PhD, COO of ���Ҵ�ý. “As we push forward with our ambitious growth strategy and work to achieve our commercial goals, their expertise will be instrumental in shaping our strategic direction.“

Laurence Reid, PhD, Chair of the Board, ���Ҵ�ý, commented: “The promise of therapies based on genome editing is fundamentally linked to understanding the desired genome modifications relative to the undesired modifications. INDUCE-seq is an extremely promising technology that will enable therapeutic developers to bring their cutting-edge treatments to market with a new standard of precision. I’m pleased to be joining the ���Ҵ�ý and eager to work closely with the leadership team to develop a comprehensive commercial strategy and support the ���Ҵ�ý in its ongoing growth.”

Brad Crutchfield, Non-Executive Director at ���Ҵ�ý, commented: “Cell and gene therapies hold immense promise for transforming disease treatment, and Broken String’s solution will be instrumental in helping companies accelerate and de-risk their drug development pipelines. By enabling the development of these next-generation therapies with precision and safety, we can enhance enterprise value for pharma and biotech firms. I look forward to leveraging my experience in bringing innovative life science tools to market to help position the company’s offerings for success in this rapidly evolving field.”

Partnership to expand applications of DNA break-mapping technology and advance understanding of genomic instability in development of the neurodegenerative disease amyotrophic lateral sclerosis

Cambridge, UK,  7 May 2024: ���Ҵ�ý (“Broken String”), a genomics company driving development of the next generation of more precise, safe, and effective cell and gene therapies, today announced it has entered a research collaboration with the Francis Crick Institute, a world-leading biomedical discovery institute dedicated to understanding the biology underlying health and disease.

In partnership with leading researchers at the Crick, the project aims to develop novel applications for Broken String’s proprietary DNA break-mapping platform, INDUCE-seq™, beyond its established capabilities in gene-editing. The research will be focused on leveraging the technology to investigate the impact of genomic instability in the development of amyotrophic lateral sclerosis (ALS). ALS is a progressive and debilitating neurodegenerative disease, causing gradual loss of the ability to control voluntary movements and basic bodily functions.

The collaboration is focused on understanding the contribution of genome stability to ALS, combining the interests of Prof Simon Boulton and Dr Nishita Parnandi at the Crick focused on genome stability and DNA double-strand break (DSB), with Prof Rickie Patani and Dr Giulia Tyzack, interested in understanding the underlying mechanism of ALS disease mechanism. Recognizing the utility of the novel INDUCE-seq platform developed by Broken String’s R&D team, led by Professor Simon Reed, the Crick and Broken String teams aim to collaborate to demonstrate and further validate the INDUCE-seq technology in this setting.

The majority of ALS cases (~90%) are considered sporadic1. Whilst there has been progress to better understand the genes and biological markers associated with the disease, very little is understood about the causes, with current treatment strategies focused on symptom management and slowing disease progression. Combining world-leading research from the Crick with Broken String’s expertise in genomics, sequencing, and bioinformatics, the partnership provides a unique opportunity to expand application of the ���Ҵ�ý’s INDUCE-seq technology in a key area of clinical unmet need, to support improved diagnosis and treatment of ALS. 

The partnership has been secured via the Francis Crick Institute’s Business Engagement Fund, a new initiative supported by The Medical Research Council (MRC-UKRI), that is designed to encourage collaborations with small-to-medium sized enterprises (SMEs) and strengthen the Crick’s engagement with industry.

Dr. Simon Boulton, Principal Group Leader, the Boulton Lab (DSB Repair Metabolism) at the Francis Crick Institute, said: “Our research is focused on exploring how cells repair damage to their DNA, and how failures in this process lead to disease. Following exploratory work with Professor Reed, we were keen to collaborate with Broken String. We are excited to leverage the INDUCE-seq platform’s unique capabilities in directly measuring and quantifying DNA double-strand breaks, and applying this to deepen our understanding of diseases that have genomic instability as a contributing factor, such as ALS.”

Felix Dobbs PhD, COO, ���Ҵ�ý, commented: “This collaboration with the Crick Institute is validation of our differentiated approach to DNA break-mapping; enabling our team to support world-leading research with insights provided through our INDUCE-seq platform. It demonstrates a fantastic opportunity to apply our expertise across other key research areas to support the advancement of human health.” He added: “There is an unmet clinical need for effective ALS treatments, as well as strategies for earlier diagnosis that can significantly improve patient outcomes. We look forward to working closely with Dr Boulton and Professor Patani’s groups to support this critical research area and continue building out our application focuses.”