Center for
Therapeutic
Genetics

Rewriting the path from discovery to treatment and shared learnings

 

A landmark collaboration between the Broad Institute, The Jackson Laboratory, and Boston Children's Hospital, united by a single mission: to develop breakthrough genetic treatments for rare diseases and deliver them to patients through structured, repeatable, and scalable clinical pathways.

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Important Notice The Center for Therapeutic Genetics is in its founding phase. We are not yet treating patients or accepting clinical referrals. We are deeply committed to families seeking help and we will communicate openly as our programs advance.

Founding Partner Institutions

Broad Institute
Gene Editing Innovation
 
The Jackson Laboratory
Disease Modeling at Scale
 
Boston Children's Hospital
Clinical Expertise & Treatment

Our Mission

Fixing the genetic code
that underlies
rare disease

The Center for Therapeutic Genetics was founded on a bold conviction: that every genetic disease diagnosis can one day come with a genetic treatment. This is possible now that we have the tools to correct genetic errors causing rare disease. What is needed are the collaborations, systems, and infrastructure to bring genetic treatments rigorously and scalably to patients. Read the press release announcing the launch of the center.
 

"We can increasingly correct the genetic errors that cause ultra-rare disease. What we have lacked is a sustainable, scalable model to build treatments for these conditions that affect only a few people — something traditional drug development was never built to do. The Center for Therapeutic Genetics is a nonprofit effort that brings together scientists and clinicians who believe that by collaborating to develop these treatments and sharing what we learn openly, we can reach patients the commercial model cannot, and make each treatment faster, safer, and more accessible than the last. "

— Winston Yan, Director, Center for Therapeutic Genetics

We primarily use base editing and prime editing, while also engaging with the broader landscape of genetic medicines including antisense oligonucleotides (ASOs) and emerging biological and delivery platforms. Our focus is rare and ultra-rare conditions that currently have no treatments and no path forward.

Scientific Leadership

The people behind
the science

Winston Yan
WY

Center for Therapeutic Genetics

Winston Yan

Director

Dr. Yan brings together the Center's founding partners and provides the scientific and operational vision that drives the mission forward and builds a shared, scalable path from patient identification to clinical treatment. He is a co-founder of Arbor Biotechnologies, an advisor of Nome.bio, and founding president and board member of the N=1 Collaborative.

David Liu
DL

Broad Institute

David Liu

Gene Editing Innovation

A pioneering biochemist, Dr. Liu invented base editing and prime editing, which correct disease-causing mutations with single-nucleotide precision. He is a co-founder and consultant for Beam Therapeutics, Pairwise Plants, Exo Therapeutics, Chroma Medicine, and Nvelop Therapeutics. He is co-founder of Prime Medicine.

Wendy Chung
WC

Boston Children's Hospital

Wendy Chung

Clinical Genetics & Rare Disease

A clinician-scientist with deep expertise in rare disease, Dr. Chung leads clinical deployment of genetic newborn screening and ensures that every CTG program is grounded in the clinical needs of the patients it aims to serve. She is on the board of directors of Prime Medicine.

Tim Yu
TY

Boston Children's Hospital

Tim Yu

Individualized Medicine Pioneer

Dr. Yu is a neurologist and researcher at Boston Children’s Hospital in the Division of Genetics & Genomics. Dr. Yu is recognized as a pioneer in individualized genetic therapeutics. He is co-founder and board member of the N=1 Collaborative.

Cat Lutz
CL

The Jackson Laboratory

Cat Lutz

Preclinical Pipeline & Translational Research

A leader in rare disease model development and translational science, Dr. Lutz directs the preclinical infrastructure that moves therapeutic candidates from early validation through the in vivo evidence packages required for IND submission.

01

Building for Many, Not One

Most rare disease treatments are developed one at a time, for one condition at a time. We are building differently. Central to the CTG model is a platform strategy, in which design tools, disease models, manufacturing processes, safety data, and clinical protocols developed for one program are shared across multiple disease programs. Each program improves the infrastructure and best practices to benefit the next. Furthermore, CTG will openly share validated procedures and data to enable institutions worldwide. The result, over time, is a flywheel: a scientific and regulatory infrastructure that improves with each treatment developed, making genetic medicine faster, safer, and less costly.

02

Regulatory Partnership

Bringing these treatments to patients will require regulatory frameworks suited to medicines that are, by design, made for one person or a few. CTG intends to work alongside the FDA and other authorities to generate the kind of rigorous evidence that can inform the evolution of such frameworks. CTG partners with industry, non-profits, and rare disease patient advocacy groups, and actively seeks new collaborations.

03

Certified Clinical Pathway

When a child is born with a serious heart defect, a specialized surgical team mobilizes to correct it, using procedures refined over decades into safe, repeatable protocols. For a child born with a serious genetic condition, no comparable path yet exists. CTG aspires to change that. Our goal is a future in which patient-tailored genetic treatments are as precise, reliable, and repeatable as today's most complex medical procedures available at scale, and sustainably available for those who will benefit from the treatment.

04

Our Science

Our genetic treatments will target the precise location in a person's DNA where a misspelling is known to cause genetic disease, and correct it. We use base and prime editors, technologies pioneered by our founding partner at the Broad Institute, to precisely change or replace the specific sequences responsible for disease. Unlike earlier gene therapies that add replacement copies of genes to cells, base- and prime-editing tools rewrite the DNA directly, addressing the root cause rather than compensating for the DNA misspelling.

When such editors are combined with advances in delivery (such as lipid nanoparticle and viral vectors), these platforms allow us to reach tissues, including the central nervous system, liver, muscle, retina, and cells in blood that were inaccessible to medicine even a decade ago.

Beyond editing, antisense oligonucleotide (ASO) therapy offers a complementary path. ASOs are short, chemically synthesized strands of nucleic acid designed to bind messenger RNA and modulate how a gene's instructions are read. They can silence toxic transcripts, or alter splicing to restore healthy RNA expression, enabling precise treatment of amenable genetic diseases.

05

Openness

From the start, CTG plans to publish its methods and share validated data and safety findings, so that a process built or a problem solved for one patient informs the treatment odyssey and eases the road for others. Additionally, a core part of our mission is to train the next generation of clinicians and scientists to scale the field of Interventional Genetics.

CTG begins small and expects the work to take years. It is built to grow and hopes to welcome additional institutions and partners as it matures.
The phases below reflect where we are today and the path forward as therapeutic programs mature.

● Current Stage
Phase 01  ·  Foundation
Institutional Formation & Scientific Planning

The Center is being formally established. Scientific leadership, governance structures, and partner coordination are underway. We are identifying priority disease areas for development. Alongside this scientific groundwork, we are actively engaged in fundraising and philanthropic outreach to secure the resources needed to move our earliest programs into preclinical development. We welcome partnerships with manufacturing and genetic treatment delivery organizations aligned with our mission.

Phase 02  ·  Preclinical
Target Validation & IND Preparation

Rigorous laboratory and animal-model studies will validate therapeutic candidates. Manufacturing processes will be developed and optimized to ensure consistent, clinical-grade production. Formal toxicology studies will establish the safety profile required for regulatory submission. Platform IND filings will be prepared in collaboration with regulatory affairs specialists and FDA scientific liaisons.

Future  ·  Phase 03  ·  First-in-Human
Clinical Trial Initiation

Initial clinical programs will open under INDs at certified clinical sites for safety monitoring, dose escalation, and evaluation of early efficacy signals.

Future  ·  Phase 04  ·  Expansion
Broader Access & New Disease Programs

Validated platform technologies will be extended to additional rare disease indications, with the goal of creating a sustainable model for therapeutic genetics at scale.

The flywheel effect

Each program generates platform knowledge, safety data, delivery validation, and regulatory precedent that directly accelerates the next. Over time, the cycle iterates and accelerates, reaching more diseases faster while maintaining scientific rigor.

Center for Therapeutic Genetics flywheel A circular flywheel showing iterative platform learning across program cycles. Preclinical Model build & validation IND & trial Platform submission Clinical learning Safety & efficacy data iterative platform learning

By approaching genetic therapies not as products, but as a clinical process, and sharing what we learn openly, we enable institutions everywhere to deliver precision genetic medicine that is faster, safer, and more accessible.

Disease Programs

From discovery
to treatment

The Center for Therapeutic Genetics will initially focus on base editing and prime editing, building each one through a rigorous preclinical pipeline before advancing toward clinical application.

Current programs
In Development — Preclinical

Liver Gene Editing via Lipid Nanoparticle Delivery

Our lead editing program targets monogenic liver diseases through systemic delivery of gene editors encapsulated in lipid nanoparticles (LNP). LNP-mediated delivery to hepatocytes has emerged as one of the most clinically validated delivery routes in genetic medicine, and we are applying this platform to diseases where a single corrective edit in liver tissue can profoundly improve serious genetic disease.

In Development — Preclinical

Central Nervous System Program

A second set of programs targeting rare monogenic diseases of the central nervous system (CNS) is under development. The CNS presents distinct delivery challenges and requires specialized in vivo validation, areas where our preclinical infrastructure is actively building capability. This work is supported by the ARPA-H THRIVE program, and further details will be announced as the program advances through scientific review.

Research infrastructure

Preclinical Infrastructure

Every program we advance is built on a foundation of rigorous preclinical science. Currently, we develop and validate both in vitro and in vivo models for each disease we pursue, ensuring that therapeutic candidates are thoroughly characterized before any consideration of clinical translation.

In the laboratory, cell-based systems allow us to confirm target biology, test editing or ASO strategies, and assess early efficacy in the precise genetic context of the disease. These findings are then further evaluated in living organisms through genetically-defined animal models, where we measure biodistribution, target engagement, durability of effect, and safety. If the right model does not yet exist, we build one.

This end-to-end preclinical pipeline spanning cellular validation, in vivo testing, manufacturing development, and safety studies produces the data packages required for regulatory submission. This is the scientific foundation for every CTG program.

Questions & Answers

Not yet. We are currently in the founding and preclinical phase. No clinical programs are open at this time. We are building toward clinical trials with urgency. Please sign up for email updates (link to subscribe form) to be notified the moment that changes.

Our initial focus is on rare and ultra-rare genetic diseases — conditions caused by pathogenic genetic variants that can be corrected with precision gene editing tools such as base and prime editors, or RNA therapies such as antisense oligonucleotides (ASOs). We will announce specific disease programs as they advance through our scientific review process.

An Investigational New Drug (IND) application is the regulatory filing required by the FDA before a new treatment can be tested in humans. Under CTG’s proposed “platform IND” strategy, safety and delivery data established for one treatment can be applied across treatments for other diseases, dramatically reducing the time and cost of bringing each treatment to patients. This approach, supported by the FDA through its rare disease and platform technology frameworks, is central to how we plan to move quickly while maintaining rigorous standards.

We read every message and we are deeply grateful for your interest. We are working hard to establish the Center for Therapeutic Genetics and our first therapeutic programs, so we may be unable to respond individually at this stage. Please know that your story and your disease area genuinely inform our scientific priorities. You will receive updates from us as our programs progress.

Most gene therapy programs are developed by individual biotechnology companies focused on a single disease or target. Economically, these organizations are constrained in the disease populations for which they can develop treatments. The Center for Therapeutic Genetics operates as a nonprofit effort designed to share platform infrastructure across many rare diseases, making it economically and operationally viable to address conditions that a commercial company could not justify pursuing alone.

Although we cannot predict exactly when gene editing will be available for each rare disease, we are encouraged by the fact that 20+ clinical trials using base editing or prime editing are currently underway with some in advanced clinical trial stages. This provides the scientific and regulatory foundations for our efforts to treat underserved, ultra-rare disease populations as a nonprofit effort. While there are still important issues to resolve, we are building the infrastructure to accelerate that timeline responsibly.

The following resources provide reliable information about how clinical trials work and how to search for ones that may be relevant to your situation.

Learn about clinical trials

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