CRISPR Clinical Trials: How to Find and Enroll in a Gene Editing Study

If you or someone you love has a genetic disease, you may have heard that CRISPR clinical trials are happening right now. That is true. As of early 2026, more than 250 clinical trials involving gene editing technologies are registered worldwide, and many of them are actively recruiting patients. Some of these trials are for conditions that had no effective treatment five years ago.

But knowing that trials exist and knowing how to actually find, evaluate, and enroll in one are very different things. The process can feel overwhelming. Trial listings are written in clinical jargon. Eligibility criteria are dense and specific. Some trials are listed as "recruiting" but are effectively full. And patients who are already dealing with the burden of a serious illness are expected to navigate this system largely on their own.

This guide is designed to change that. We will walk through exactly how to search for gene editing clinical trials, explain what the different phases mean for you as a patient, describe what participation actually involves from start to finish, highlight specific CRISPR trials that are currently recruiting, and connect you with organizations that can help. The goal is to give you clear, honest, actionable information so you can make an informed decision about whether a clinical trial might be right for you.

Understanding Clinical Trials: What They Are and What They Are Not

A clinical trial is a structured research study that tests whether a treatment is safe and effective in people. For gene editing therapies, clinical trials are the path between the laboratory and the pharmacy. Every CRISPR therapy that eventually reaches patients — Casgevy for sickle cell disease, for example — went through this process first.

Clinical trials are not a last resort. They are not a sign that your doctor has given up. In many cases, clinical trials offer access to therapies that are years ahead of anything commercially available. Patients in CRISPR clinical trials have received treatments that, if successful, could fundamentally change the course of their disease.

That said, clinical trials are not guaranteed cures. They are experiments, and by definition, the outcome is uncertain. Some patients in trials receive the experimental treatment and experience remarkable benefits. Others receive it and see no improvement. In some trial designs, patients may receive a placebo or a standard treatment instead of the experimental therapy. Transparency about these realities is essential to making a good decision.

The Four Phases of Clinical Trials

Clinical trials for new drugs and therapies in the United States follow a structured progression overseen by the FDA. Each phase has a specific purpose and carries different implications for patients considering enrollment.

Phase 1: Is It Safe?

Phase 1 trials are the first time a therapy is tested in humans. The primary goal is to evaluate safety and determine the appropriate dose. These trials are typically small, enrolling between 10 and 30 participants. For gene editing therapies, Phase 1 trials often begin with patients who have the most severe forms of a disease, because the potential benefit must justify the unknown risks.

As a patient, enrolling in a Phase 1 trial means you are among the first people to receive a therapy. The potential upside is access to a cutting-edge treatment. The potential downside is that less is known about risks and side effects. Phase 1 trials include intensive monitoring and frequent hospital visits.

Phase 2: Does It Work?

Phase 2 trials expand the study to a larger group, typically 30 to 100 patients. The goal is to gather preliminary evidence of efficacy — does the therapy actually help? — while continuing to monitor safety. Dose adjustments may still occur. Many gene editing trials currently recruiting are in Phase 1/2, meaning they combine safety and efficacy evaluation in a single study.

Phase 3: How Does It Compare?

Phase 3 trials are large-scale studies, often involving hundreds of patients across multiple treatment centers. The therapy is compared against the current standard of care or, in some cases, a placebo. These trials generate the data that the FDA uses to make approval decisions. Phase 3 trials offer the advantage of a more established safety profile, but enrollment criteria are often stricter, and the study design may include randomization — meaning you may not receive the experimental therapy.

Phase 4: Post-Market Surveillance

Phase 4 trials occur after a therapy has been approved and is on the market. They monitor long-term safety and effectiveness in a broader patient population. For gene editing therapies, Phase 4 monitoring is particularly important because the long-term effects of permanently altering DNA are still being studied.

Most CRISPR gene editing trials currently available to patients are in Phase 1, Phase 1/2, or Phase 2. This reflects the relative youth of the technology as a clinical tool. The exception is Casgevy and a few other therapies that have completed their pivotal trials and received regulatory approval.

How to Search ClinicalTrials.gov

ClinicalTrials.gov is the primary database for finding clinical trials in the United States and many international studies. It is maintained by the National Library of Medicine at the U.S. National Institutes of Health. Every clinical trial conducted in the United States is legally required to be registered here.

The database contains over 500,000 studies, which can feel overwhelming. Here is a step-by-step approach to finding relevant gene editing trials.

Step 1: Start with a Simple Search

Go to ClinicalTrials.gov and use the search bar. Type your condition first, then add "CRISPR" or "gene editing" or "gene therapy." For example:

"sickle cell disease CRISPR"
"beta thalassemia gene editing"
"Duchenne muscular dystrophy gene therapy"
"cancer CRISPR"
"hereditary angioedema gene editing"

The site will return a list of studies with basic information about each.

Step 2: Use Filters to Narrow Results

After your initial search, use the filter options on the left side of the results page:

Recruitment status: Select "Recruiting" to see only trials that are actively enrolling patients right now. You can also select "Not yet recruiting" to see trials that are expected to open soon.
Phase: Filter by Phase 1, Phase 2, or Phase 3 depending on your comfort level.
Age group: Trials specify whether they accept children, adults, or older adults.
Location: Filter by country, state, or city to find trials near you.
Study type: Select "Interventional" to see treatment trials rather than observational studies.

Step 3: Read the Study Record Carefully

Each trial listing on ClinicalTrials.gov includes several critical sections:

Brief Summary: A plain-language description of the study's purpose.
Eligibility Criteria: The inclusion and exclusion criteria that determine whether you qualify. Read these carefully. They specify required diagnoses, age ranges, prior treatments, lab values, and medical conditions that would disqualify you.
Contacts and Locations: The treatment sites and the people you can contact to inquire about enrollment. This is your direct line to the research team.
Study Design: Details about randomization, blinding, and what treatments are being compared.
Outcome Measures: What the researchers are measuring to determine if the therapy works.

Step 4: Contact the Research Team

If a trial looks like a potential fit, contact the study coordinator listed in the trial record. Do not wait for a doctor's referral. You can call or email directly. The coordinator will ask some preliminary questions about your medical history to determine if you might be eligible for a screening visit.

Beyond ClinicalTrials.gov

While ClinicalTrials.gov is the most comprehensive database for U.S. trials, several other resources can help:

WHO International Clinical Trials Registry Platform (ICTRP): Aggregates trial registries from around the world, useful if you are open to trials outside the United States.
EU Clinical Trials Register: The European equivalent for trials conducted in EU member states.
Company websites: Biotech companies running CRISPR trials (Intellia Therapeutics, Beam Therapeutics, Editas Medicine, Verve Therapeutics, CRISPR Therapeutics) often have dedicated patient pages with information about their active studies, written in more accessible language than ClinicalTrials.gov.
Patient advocacy organization trial finders: Organizations like the Sickle Cell Disease Association of America, Parent Project Muscular Dystrophy, and the National Organization for Rare Disorders maintain curated lists of relevant trials for their specific disease communities.

CRISPR and Gene Editing Trials Currently Recruiting

The landscape of gene editing clinical trials is evolving rapidly. The following represents a snapshot of notable trials that were recruiting or expected to begin recruitment as of early 2026. We recommend verifying current status on ClinicalTrials.gov, as enrollment windows can open and close quickly.

Intellia Therapeutics — NTLA-2002 for Hereditary Angioedema (HAELO Trial)

Intellia's HAELO program is one of the most closely watched CRISPR trials in the world. NTLA-2002 is an in vivo CRISPR therapy delivered directly into the bloodstream via lipid nanoparticles. It targets the KLKB1 gene in the liver to reduce production of kallikrein, the protein responsible for the painful and potentially life-threatening swelling episodes that characterize hereditary angioedema (HAE).

What makes HAELO remarkable is that it is a one-time intravenous infusion — no surgery, no stem cell transplant, no conditioning chemotherapy. In Phase 1/2 data reported in 2025, patients experienced a greater than 95 percent reduction in swelling attacks, with some patients becoming completely attack-free. Intellia's Phase 3 HAELO trial is currently enrolling patients with Type I or Type II HAE at sites across the United States, Europe, and other regions.

If you have HAE: Visit Intellia's patient page or search ClinicalTrials.gov for "NTLA-2002" to find the nearest trial site.

Beam Therapeutics — BEAM-101 for Sickle Cell Disease

Beam Therapeutics is developing BEAM-101, a base editing therapy for sickle cell disease. Unlike traditional CRISPR-Cas9, which cuts both strands of DNA, base editing makes precise single-letter changes without creating double-strand breaks. BEAM-101 works by editing the patient's own blood stem cells to reactivate fetal hemoglobin, which compensates for the defective adult hemoglobin that causes red blood cells to sickle.

The BEACON trial (Phase 1/2) is evaluating BEAM-101 in patients aged 18 to 35 with severe sickle cell disease. Like Casgevy, it is an ex vivo therapy — the patient's stem cells are collected, edited in a laboratory, and then reinfused after conditioning chemotherapy. Early data from the first treated patients have shown promising increases in fetal hemoglobin levels.

Search ClinicalTrials.gov for "BEAM-101" or visit Beam Therapeutics' website for enrollment information.

Verve Therapeutics — VERVE-102 for Familial Hypercholesterolemia

Verve Therapeutics is pioneering in vivo base editing for cardiovascular disease. VERVE-102 targets the PCSK9 gene in the liver to permanently lower LDL cholesterol in patients with heterozygous familial hypercholesterolemia (HeFH), a genetic condition affecting roughly 1 in 250 people that causes dangerously elevated cholesterol from birth and dramatically increases heart attack risk.

The Heart-2 trial is currently enrolling patients with HeFH who have atherosclerotic cardiovascular disease despite maximum tolerated lipid-lowering therapy. Like the Intellia approach, this is delivered as a single intravenous infusion. If successful, it could replace a lifetime of daily medication with one treatment.

Editas Medicine — EDIT-301 for Sickle Cell Disease

Editas Medicine is developing EDIT-301, another gene editing therapy for sickle cell disease that uses a high-fidelity Cas12a enzyme (rather than the Cas9 used in Casgevy). The therapy edits the HBG1/2 promoter region to reactivate fetal hemoglobin production. The EdiTHAL trial is enrolling patients with severe sickle cell disease, and early clinical data have shown robust fetal hemoglobin induction.

Excision BioTherapeutics — EBT-101 for HIV

One of the most ambitious applications of CRISPR is the attempt to cure HIV by excising integrated viral DNA from the genomes of infected cells. EBT-101 uses a multiplexed CRISPR approach with multiple guide RNAs to cut the HIV provirus at several locations, delivered via an AAV9 vector. The Phase 1/2 trial is ongoing and represents the first in vivo CRISPR therapy aimed at targeting a viral genome within a living person.

CRISPR Therapeutics — CTX Series for Cancer

CRISPR Therapeutics, the company behind Casgevy, is also running several trials of CRISPR-edited CAR-T cell therapies for cancer. Their CTX110, CTX112, and CTX131 programs use CRISPR to create "off-the-shelf" allogeneic CAR-T cells — immune cells that can be manufactured from healthy donors and given to any patient, rather than requiring individualized manufacturing from each patient's own cells.

Trials for CTX112 (targeting CD19 for B-cell malignancies) and CTX131 (targeting CD70 for renal cell carcinoma) have been recruiting at oncology centers in the United States. These represent an important evolution in cancer immunotherapy, potentially making CAR-T treatment faster, cheaper, and more widely accessible.

What Does Participation Actually Involve?

Understanding the practical reality of clinical trial participation is essential before you make a decision. Here is what to expect.

Screening and Eligibility Determination

After you contact a trial site, the first step is a screening process. This typically involves a detailed review of your medical records, a physical examination, blood tests, and sometimes imaging studies or genetic testing. The purpose is to determine whether you meet the eligibility criteria. Screening can take one to several weeks.

Not everyone who wants to participate will qualify. Eligibility criteria exist to protect patient safety and ensure the study can generate meaningful data. Common reasons people are excluded include having certain other medical conditions, being on medications that could interfere with the therapy, not meeting specific lab value thresholds, or being outside the specified age range. Being excluded does not mean you are out of options — it means that specific trial was not the right fit, and the research team may be able to suggest alternatives.

Informed Consent

Before any study procedures begin, you will go through an informed consent process. This is not just signing a form. A member of the research team will explain, in detail:

The purpose of the study
What the experimental treatment is and how it works
All known risks and potential side effects
What procedures you will undergo and how often
Whether there is a chance you will receive a placebo or standard treatment instead
Your right to withdraw from the study at any time, for any reason, without it affecting your medical care
How your privacy and medical information will be protected

You should take as much time as you need to read the consent document. Bring it home. Discuss it with your family. Ask every question that comes to mind. The research team is required to answer all your questions honestly and thoroughly. If you feel pressured to sign, that is a red flag.

The Treatment Phase

What happens next depends on the type of therapy and the trial design.

For ex vivo gene editing trials (like BEAM-101 or EDIT-301 for sickle cell disease): Your blood stem cells are collected through a process called apheresis, similar to blood donation. The cells are then shipped to a manufacturing facility where CRISPR editing occurs. This manufacturing process takes several weeks. Before you receive the edited cells back, you will undergo conditioning chemotherapy — typically with busulfan — to make room in your bone marrow for the edited cells. The conditioning regimen is one of the most physically demanding parts of the process, with side effects that can include nausea, fatigue, low blood counts, and temporary immune suppression. After the edited cells are infused, you will be hospitalized for monitoring, usually for four to six weeks.

For in vivo gene editing trials (like Intellia's NTLA-2002 or Verve's VERVE-102): The treatment is typically a single intravenous infusion administered in a clinical setting. No surgery, no stem cell collection, no conditioning chemotherapy. The infusion itself may take an hour or two. You will be monitored for several hours afterward and may stay overnight. The recovery period for in vivo therapies is generally much shorter and less physically demanding than for ex vivo approaches.

Follow-Up and Monitoring

All gene editing clinical trials require extensive follow-up. This is not optional. Because gene editing permanently alters DNA, regulators require long-term safety monitoring.

Expect follow-up visits at regular intervals — weekly at first, then monthly, then quarterly, then annually. These visits typically involve blood draws, physical examinations, and assessments of how you are responding to the therapy. Many trials require follow-up for at least five years, and some require 15 years of monitoring.

This is a significant commitment, and you should factor it into your decision. If you live far from the trial site, the travel burden can be substantial.

Risks and Side Effects

Every clinical trial carries risk. For gene editing therapies, the risks fall into several categories:

Therapy-specific risks: Off-target DNA editing (cutting at unintended genomic locations), on-target but unintended effects (such as large deletions at the editing site), and immune reactions to the delivery vehicle (lipid nanoparticles or viral vectors).
Conditioning chemotherapy risks (for ex vivo trials): Bone marrow suppression, infection, infertility, and in rare cases, secondary cancers. The conditioning regimen is often the highest-risk component of ex vivo gene editing trials. Patients should be counseled about fertility preservation options before beginning conditioning.
Unknown long-term risks: Because gene editing permanently changes DNA, there are theoretical concerns about effects that might not manifest for years or decades. This is why long-term follow-up is required.
Psychological and emotional risks: The uncertainty of participating in a trial, the physical demands of treatment, time away from work and family, and the possibility that the therapy may not work can take a significant emotional toll.

You should discuss all of these risks thoroughly with the research team and your own physician before deciding to participate.

The Financial Reality: Clinical Trials Are Usually Free

One of the most important and least understood aspects of clinical trial participation is cost. In the vast majority of gene editing clinical trials, the experimental therapy itself is provided at no cost to the patient. The pharmaceutical company sponsoring the trial covers the cost of the drug, the manufacturing, and the study-related procedures.

However, "free" has important caveats:

Standard-of-care costs: Medical procedures that would be part of your normal care regardless of the trial (routine blood tests, doctor visits for your underlying condition) may still be billed to your insurance. The line between "study-related" and "standard care" can be blurry, and it varies by trial.
Travel and lodging: Many trials require you to travel to a specialized treatment center, sometimes in another city or state. Some but not all trials offer travel stipends or reimbursement. Ask about this upfront.
Lost wages: Time away from work for treatment and follow-up visits is a real cost. Some employers are accommodating; others are not. The Family and Medical Leave Act (FMLA) may protect your job during extended treatment, but it does not guarantee paid leave.
Insurance concerns: Federal law (specifically, the Affordable Care Act, Section 2709) requires most health insurers to cover routine patient costs during clinical trials. This means your insurance generally cannot refuse to cover normal medical costs simply because you are in a trial. However, enforcement and interpretation vary, and disputes do occur.

Many trial sponsors also offer patient support programs that help with logistics, travel, housing near the treatment center, and navigating insurance questions. Ask the study coordinator what support is available.

Patient Advocacy Organizations That Can Help

You do not have to navigate this alone. Several organizations specialize in connecting patients with clinical trials and providing support throughout the process.

National Organization for Rare Disorders (NORD)

NORD maintains a comprehensive database of rare disease clinical trials and offers patient assistance programs, educational resources, and a helpline. If you have a rare genetic condition, NORD should be one of your first stops. They also offer co-pay assistance for approved therapies and travel grants for clinical trial participants.

Parent Project Muscular Dystrophy (PPMD)

PPMD is the leading advocacy organization for Duchenne muscular dystrophy. They maintain a detailed clinical trial finder specific to DMD, offer educational webinars on trial participation, and provide one-on-one support through their Decode Duchenne program, which includes genetic testing that can help determine trial eligibility.

Sickle Cell Disease Association of America (SCDAA)

SCDAA provides resources specifically for sickle cell patients considering gene therapy clinical trials. They maintain a clinical trial navigator, host community education events, and advocate for equitable access to emerging therapies. Given that several CRISPR trials are targeting sickle cell disease, SCDAA is an essential resource.

CureSMA

CureSMA supports families affected by spinal muscular atrophy and provides trial matching services, educational materials, and community support.

The Cystic Fibrosis Foundation

The CF Foundation offers a clinical trial finder, partners directly with pharmaceutical companies on trial design, and provides financial assistance for trial participants.

Genetic Alliance

Genetic Alliance is a network of more than 10,000 organizations that connects patients with genetic conditions to resources, including clinical trials. Their platform can help you find disease-specific organizations relevant to your condition.

The Leukemia & Lymphoma Society (LLS)

For patients with blood cancers who may be eligible for CRISPR-edited CAR-T cell trials, LLS offers clinical trial support services including a dedicated nurse navigator who can help identify appropriate trials.

Questions to Ask Your Doctor

Before enrolling in any clinical trial, have a detailed conversation with your physician. Here are specific questions that will help you evaluate whether a trial is right for you:

About the trial itself:

What is the specific gene editing technology being used, and how does it differ from other available options?
What phase is this trial? What has been learned from earlier phases?
Is there a chance I will receive a placebo instead of the experimental treatment?
How many patients have been treated so far, and what results have been reported?
Who is the sponsor, and what is their track record?

About eligibility and your condition:

Based on my specific genetic mutation and medical history, am I likely to be eligible?
Are there other trials I should also consider?
Would participating in this trial prevent me from being eligible for other trials or treatments in the future?
How does the potential benefit of this trial compare to my current treatment plan?

About logistics and risks:

Where is the treatment center, and how long will I need to be there?
What is the most serious risk associated with this therapy?
What are the side effects of the conditioning regimen, if applicable?
Should I pursue fertility preservation before treatment?
What happens if the therapy does not work? What are my options afterward?
What is the follow-up schedule, and how long will monitoring continue?

About costs:

Will all study-related costs be covered, or will some procedures be billed to my insurance?
Does the sponsor offer travel and lodging assistance?
Are there patient advocacy organizations that can provide financial support?

Your doctor may not know the answers to all of these questions, particularly about specific trial protocols. That is okay. The study coordinator at the trial site can fill in the details. The point is to have an honest, thorough conversation before making a commitment.

Common Misconceptions About Clinical Trials

Several persistent myths discourage patients from exploring clinical trials. Let us address them directly.

"Clinical trials are a last resort." This is one of the most harmful misconceptions. Many gene editing trials specifically seek patients who are early in their disease course, precisely because the therapy may be more effective before significant organ damage has occurred. Some trials require that patients have not received certain prior treatments. Waiting too long can actually disqualify you.

"I might get a sugar pill." Placebo-controlled trials do exist, but they are less common in gene editing studies than many people assume. Most current CRISPR trials use open-label designs (everyone knows what they are receiving) or compare the experimental therapy against the current standard of care rather than a placebo. When placebo controls are used, it is typically only for a defined period, after which all participants may receive the experimental therapy (a crossover design). The informed consent document will tell you exactly what the trial design is.

"I will be a guinea pig." Clinical trials are among the most heavily regulated activities in medicine. Every trial must be approved by an Institutional Review Board (IRB), an independent committee that evaluates the study's design, risks, and informed consent process. The FDA monitors ongoing trials through required safety reports. Participants have the right to withdraw at any time. And the research team has a legal and ethical obligation to stop the trial if unexpected safety signals emerge. This does not mean trials are risk-free, but it does mean they are not unregulated experiments.

"Clinical trials are only for people with no other options." Many trial participants have good existing treatment options but choose to participate because the experimental therapy offers the possibility of a better outcome. Patients with sickle cell disease who are managing their condition with hydroxyurea, for example, may choose to enroll in a CRISPR trial because a one-time gene editing treatment could eliminate the need for lifelong medication and the ongoing risk of vaso-occlusive crises.

"I cannot afford to participate." As discussed above, the experimental therapy is almost always free. While there are real costs associated with travel and time, many trials offer support, and advocacy organizations can help fill gaps.

Special Considerations for Pediatric Patients

Many genetic diseases manifest in childhood, and parents naturally want to know whether clinical trials are available for their children. Gene editing trials for pediatric patients do exist, but they raise additional considerations.

Regulatory agencies require that adult safety data be established before trials extend to children. This means pediatric trials often lag behind adult trials by several years. However, some conditions — like sickle cell disease, beta thalassemia, and Duchenne muscular dystrophy — primarily affect children and young adults, and regulatory agencies have provided pathways to include younger patients when the risk-benefit calculation supports it.

For pediatric trials, both parental consent and, when age-appropriate, the child's assent are required. The IRB applies additional protections for minor participants. If your child has a genetic condition and you are interested in trial options, your pediatrician or genetic counselor can help you identify age-appropriate studies, and disease-specific advocacy organizations like PPMD and CureSMA maintain pediatric-specific resources.

A Practical Checklist for Getting Started

If you are considering a clinical trial, here is a concrete plan of action:

Get your genetic testing results. Many gene editing trials require a confirmed genetic diagnosis with a specific mutation identified. If you have not had genetic testing, ask your doctor to order it or contact a genetic counselor. Some advocacy organizations, like PPMD's Decode Duchenne program, offer free genetic testing.
Search ClinicalTrials.gov. Use your condition name and terms like "CRISPR," "gene editing," "gene therapy," or "base editing." Filter for "Recruiting" status and your geographic area.
Check company websites. Visit the patient sections of Intellia, Beam, Verve, Editas, CRISPR Therapeutics, and other gene editing companies to see their active trials in patient-friendly language.
Contact a patient advocacy organization. They can help you navigate the search process, understand eligibility criteria, and connect you with other patients who have been through the experience.
Talk to your doctor. Share what you have found and ask for their assessment of whether a trial is appropriate for your situation.
Contact the trial site directly. Call or email the study coordinator. Ask your preliminary questions. If you seem like a potential fit, they will guide you through the screening process.
Take your time with informed consent. Do not rush. Read everything. Ask every question. Involve your family.
Plan for logistics. Understand the time commitment, travel requirements, and financial considerations before you enroll.

The Bigger Picture

We are living through a period in medicine that will be studied for centuries. The ability to precisely edit the human genome — to correct the misspellings in DNA that cause disease — was science fiction fifteen years ago. Today it is happening in hospitals and treatment centers around the world. Casgevy has already cured patients of sickle cell disease. Intellia's one-time infusion has eliminated angioedema attacks. Beam's base editing approach is showing early promise of doing the same without double-strand DNA breaks.

But the gap between scientific possibility and patient access remains wide. Clinical trials are the bridge. They are the mechanism through which experimental therapies become proven treatments, and they depend on the willingness of patients to participate. Every person who enrolls in a gene editing trial contributes not only to their own potential treatment but to the knowledge base that will help future patients.

If you are considering this path, know that you are not alone, that there are people and organizations who will help you navigate the process, and that the decision to participate — or not to participate — is entirely yours to make, on your own terms, with complete information.

Sources

U.S. National Library of Medicine. ClinicalTrials.gov. https://clinicaltrials.gov/
Intellia Therapeutics. NTLA-2002 Phase 3 HAELO Trial for Hereditary Angioedema. Clinical data presentations, 2025.
Beam Therapeutics. BEAM-101 BEACON Trial for Sickle Cell Disease. https://beamtx.com/
Verve Therapeutics. VERVE-102 Heart-2 Trial for Heterozygous Familial Hypercholesterolemia. https://www.vervetx.com/
Editas Medicine. EDIT-301 EdiTHAL Trial for Sickle Cell Disease. https://www.editasmedicine.com/
Excision BioTherapeutics. EBT-101 for HIV Cure. Phase 1/2 clinical trial data. https://www.excision.bio/
CRISPR Therapeutics. CTX110, CTX112, and CTX131 Allogeneic CAR-T Programs. https://crisprtx.com/
National Organization for Rare Disorders (NORD). Clinical Trial Resources. https://rarediseases.org/
Parent Project Muscular Dystrophy (PPMD). Decode Duchenne and Clinical Trial Finder. https://www.parentprojectmd.org/
Sickle Cell Disease Association of America (SCDAA). Gene Therapy Resources. https://www.sicklecelldisease.org/
U.S. Food and Drug Administration. "FDA Guidance for Clinical Trial Sponsors: Informed Consent." https://www.fda.gov/
Affordable Care Act, Section 2709. Coverage of Routine Patient Costs in Clinical Trials. Public Law 111-148.
World Health Organization. International Clinical Trials Registry Platform (ICTRP). https://www.who.int/clinical-trials-registry-platform
Henderson, G.E., et al. "Clinical Trials and Medical Care: Defining the Therapeutic Misconception." PLOS Medicine 4, no. 11 (2007): e324.
Frangoul, H., et al. "CRISPR-Cas9 Gene Editing for Sickle Cell Disease and Beta-Thalassemia." New England Journal of Medicine 384, no. 3 (2021): 252-260.