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	<title>Rett Syndrome Research Trust News</title>
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	<description>News brought to you by Rett Syndrome Research Trust</description>
	<pubDate>Thu, 18 Jun 2026 21:06:15 -0500</pubDate>
	<copyright>Copyright Rett Syndrome Research Trust</copyright>
	<ttl>60</ttl>
	<item>
		<title>A Single Shot That Rewrites the Code: What Intellia&#x00027;s CRISPR Breakthrough Means for Rett Families</title>
		<description><![CDATA[Last week, a biotech company called Intellia Therapeutics announced positive results from a large clinical trial of a CRISPR-based gene editing therapy. Intellia&#x00027;s therapy, called lonvoguran ziclumeran (lonvo-z), was tested in people with hereditary angioedema (HAE), a rare genetic condition that causes recurrent swelling attacks in the face, airway, abdomen, and limbs.

HAE is caused by overactivity of a protein called kallikrein, which drives swelling attacks. Lonvo-z uses CRISPR gene editing to permanently inactivate the gene (KLKB1) that produces kallikrein, with the goal of stopping attacks at their source. The therapy is delivered as a single intravenous infusion using lipid nanoparticles, which are small fat-based particles, similar to those used in the COVID vaccines. The lipid nanoparticles carry the CRISPR machinery into liver cells, where the editing takes place.]]></description>
		<pubDate>Thu, 07 May 2026 08:29:30 -0500</pubDate>
		<link>https://reverserett.org/news/articles/what-intellias-breakthrough-means-for-rett/</link>
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	<item>
		<title>Herbivorous Dinosaur Seeking Connection</title>
		<description><![CDATA[My daughter, Eleanor, was diagnosed with Rett syndrome when she was two-and-a-half years old. I was 43. Eleanor is now almost 17. I&#x00027;ll let you do the math on my age. I&#x00027;m not quite a dinosaur, I think.

When I started my job as chief development officer at RSRT in 2013, Facebook dominated social media, AI was largely unheard of, and emailing or calling someone on the phone was the preferred way to communicate. A lot has changed, I know. And I confess that email and phone remain the way I do most of my communicating. Okay, maybe I&#x00027;m a dinosaur.

One thing hasn&#x00027;t changed for me, though.

In 2013 I believed that the genetic-based research that RSRT is pushing forward will change Eleanor&#x00027;s life and hundreds of thousands of other lives. Today, I believe that more than ever. It&#x00027;s more than a belief. It&#x00027;s a conviction.

That conviction is based on what I see every day — the dedication, passion, expertise, energy, and wisdom of Monica and our RSRT science team in action for all with Rett syndrome.]]></description>
		<pubDate>Wed, 08 Apr 2026 11:26:33 -0500</pubDate>
		<link>https://reverserett.org/news/articles/herbivorous-dinosaur-seeking-connection/</link>
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		<title>2025 Research Awards: $6.4 Million to Advance Genetic Medicines for Rett</title>
		<description><![CDATA[Every dollar raised for RSRT is put to work on behalf of the community we serve. Our 2025 research awards total $6.4 million, bringing our lifetime investment in Rett research to $92 million since 2008. That number reflects years of disciplined, strategic funding. It also reflects something more immediate: a portfolio of programs we believe can reach clinical trials in 2028.

This year&#x00027;s awards span both sides of the genetic medicine equation. Cargo + Delivery = Genetic Medicines

What We Deliver

Technological breakthroughs over the last decade have opened doors that did not previously exist. We&#x00027;re advancing multiple approaches that address mutations at the DNA and RNA level. Profluent Bio is using artificial intelligence to design next-generation base editors built from the ground up for Rett, compact and precisely targeted rather than adapted from existing technology. Tacit Therapeutics is advancing RNA trans-splicing,]]></description>
		<pubDate>Wed, 01 Apr 2026 14:13:19 -0500</pubDate>
		<link>https://reverserett.org/news/articles/2025-research-awards/</link>
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	<item>
		<title>Coming Full Circle: Serendipity and Seizing the Day</title>
		<description><![CDATA[Since late last year I have had the honor and privilege of working with RSRT, first as a consultant and now as Chief Medical Officer.

RSRT is a truly exceptional organization that combines the vision, determination and organizational acumen of its founder Monica Coenraads (mother, advocate, and pioneer in foundation-driven therapeutic research for Rett) with the unparalleled scientific &#x00022;chops&#x00022; and depth of insight of top-notch scientists Bob Deans, Chief Technology Officer, and John Sinnamon, Director, Head of Research. It is a testament to their bona fides that they have been able to engage with the best minds in the field and have left no stone unturned in their quest for novel genetic medicines to cure Rett syndrome. 

On a personal note, joining RSRT exemplifies the power of serendipity and seizing opportunities when they come your way. My current engagement with RSRT arose by happenstance from a casual referral by my previous boss at Aspa Therapeutics, Adam Shaywitz,]]></description>
		<pubDate>Mon, 30 Mar 2026 18:33:09 -0500</pubDate>
		<link>https://reverserett.org/news/articles/coming-full-circle/</link>
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	<item>
		<title>Rett Syndrome Research Trust Appoints Genevieve Laforet, MD, PhD as Chief Medical Officer, Strengthening Leadership in Genetic Medicine Development</title>
		<description><![CDATA[Trumbull, CT — The Rett Syndrome Research Trust (RSRT), the organization singularly focused on curing Rett syndrome, today announced the appointment of Genevieve Laforet, M.D., Ph.D., as Chief Medical Officer. Dr. Laforet’s addition further strengthens RSRT’s leadership team as the organization advances multiple genetic medicine programs toward clinical development for Rett syndrome.

Dr. Laforet brings two decades of industry and academic experience with a focus on developing therapies for rare genetic neurological diseases. Prior to joining RSRT, she served as Chief Medical Officer at AS2Bio where she oversaw gene and cell therapy programs for Angelman syndrome. She previously spearheaded gene therapy development for Canavan disease at Aspa Therapeutics and held leadership roles in Duchenne muscular dystrophy programs at Solid Biosciences and Sarepta Therapeutics.

She earned her A.B. in Biochemical Sciences from Harvard College, her M.D. from Duke University School of Medicine, and her Ph.D.]]></description>
		<pubDate>Mon, 30 Mar 2026 19:21:53 -0500</pubDate>
		<link>https://reverserett.org/news/press-releases/rsrt-appoints-genevieve-laforet-chief-medical-officer/</link>
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		<title>NGN-401 Embolden™ Trial is Fully Enrolled</title>
		<description><![CDATA[Neurogene shared an update on their NGN-401 program for Rett syndrome, including progress from their ongoing clinical trial and what’s ahead.

Progress on the clinical trial:


	
	Current clinical trial (“Embolden”) is fully enrolled, and more than half of patients have already received the treatment.
	
	
	The company expects to finish dosing all patients by mid-2026.
	
	
	So far, the treatment has been generally well-tolerated, with no serious immune-related cases (HLH) at the tested dose.
	


 

Regulatory &#x00026; Development Milestones:


	
	The U.S. FDA gave NGN-401 Breakthrough Therapy designation, which can speed up development and review.
	
	
	Neurogene is preparing to apply for approval (a Biologics License Application, or BLA).
	
	
	Manufacturing is already aligned with commercial scale, which simplifies the approval process.

	 
	


What’s Expected in 2026:


	Finish dosing patients in the main trial (by mid-2026)
	Release updated clinical data (mid-]]></description>
		<pubDate>Tue, 24 Mar 2026 11:25:57 -0500</pubDate>
		<link>https://reverserett.org/news/articles/ngn-401-embolden-trial-is-fully-enrolled/</link>
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		<title>RNA Trans-splicing Comes of Age</title>
		<description><![CDATA[Trans-splicing is an exciting approach that RSRT is pushing forward as part of our Roadmap to Cures. I wanted to describe this promising approach to our community using a fun and accessible analogy - making a movie - that seems especially apropos given the recent Academy Awards. I hope you enjoy it and that it gives you a clear sense of how trans-splicing works. I look forward to keeping you updated in the months ahead. 

 

A quick introduction to RNA splicing

In biology, information generally flows from DNA → RNA → protein. DNA stores the instructions needed to build and maintain our cells. Individual genes are like files on a computer, each containing specific information. When a cell needs to make a protein, it first copies the information from the gene into RNA. This RNA contains the instructions for building the protein, but also extra material that needs to be removed. The process of editing out these extra pieces is called RNA splicing. A helpful analogy is the process of making movies.]]></description>
		<pubDate>Fri, 13 Mar 2026 14:16:50 -0500</pubDate>
		<link>https://reverserett.org/news/articles/rna-trans-splicing-comes-of-age/</link>
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	<item>
		<title>Rett Syndrome Research Trust Announces 2025 Research Awards to Advance Genetic Medicines</title>
		<description><![CDATA[Trumbull, CT — The Rett Syndrome Research Trust (RSRT), the organization singularly focused on curing Rett syndrome, today announced 2025 research awards totaling $6.4 million, further advancing genetic medicine programs into the clinic. RSRT’s cumulative $92 million investment in Rett research since its founding in 2008 has fundamentally reshaped the Rett research landscape—transforming a historically underfunded rare disease into a high-profile focus of genetic medicine innovation. Through sustained, strategic funding, RSRT has attracted world-class academic laboratories, launched multi-institutional consortia, catalyzed biotechnology company engagement, and accelerated the translation of Rett discoveries from basic science to clinical development.

Rett syndrome is a rare and devastating neurodevelopmental disorder caused by mutations in the MECP2 gene. After a period of relatively normal early development, affected children lose speech, purposeful hand use, and motor skills,]]></description>
		<pubDate>Thu, 12 Feb 2026 16:18:13 -0600</pubDate>
		<link>https://reverserett.org/news/press-releases/rsrt-2025-research-awards-to-advance-genetic-medicine/</link>
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	<item>
		<title>University of Rochester Study: Auditory Processing in Rett Syndrome</title>
		<description><![CDATA[The University of Rochester is conducting a research study examining how the brain processes sound and sensory information in individuals with Rett syndrome.

The study uses electroencephalography (EEG), a safe and non-invasive method of recording brain activity, to identify patterns that may help researchers better assess cognition in Rett syndrome.

Who may participate:

Individuals with Rett syndrome and a genetically confirmed MECP2 mutation may be eligible. Participants must be past the clinical regression stage, have no known hearing impairment, and have no additional neurological or genetic diagnoses beyond Rett syndrome.

What participation involves:

Participants complete an EEG session during which they listen to tones through headphones. A caregiver is also asked to complete surveys and an interview about their child’s symptoms, daily functioning, and behaviors.

Study visits last approximately 3–4 hours,]]></description>
		<pubDate>Thu, 22 Jan 2026 14:27:09 -0600</pubDate>
		<link>https://reverserett.org/news/articles/urmc-rettstudy/</link>
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	<item>
		<title>Taysha Program Update: January 2026</title>
		<description><![CDATA[Taysha Gene Therapies has shared an update on its TSHA-102 gene therapy program for Rett syndrome through a press release and a community letter. Links to both documents are available below.

Highlights include:


	
	The first participant was dosed in the REVEAL Pivotal Study in the fourth quarter of 2025. Enrollment is ongoing across multiple clinical trial sites.
	
	
	Taysha reached written alignment with the U.S. Food and Drug Administration (FDA) on a study for younger girls (ASPIRE Study).
	
	
	All participants in both the REVEAL Pivotal Study and the ASPIRE Study are expected to be dosed by the second quarter of 2026.
	
	
	Longer-term safety and efficacy data from Part A of the REVEAL Phase 1/2 study is expected in the first half of 2026.
	


 

Study overview:


	REVEAL Pivotal Study: Evaluating the safety and efficacy of TSHA-102 in 15 girls and young women ages 6 to under 22 with typical Rett syndrome.]]></description>
		<pubDate>Tue, 06 Jan 2026 09:42:39 -0600</pubDate>
		<link>https://reverserett.org/news/articles/taysha-program-update-january-2026/</link>
		<guid>https://reverserett.org/news/articles/taysha-program-update-january-2026/</guid>
	</item>
	<item>
		<title>2025 Rett Genetic Medicines Summit: Key Takeaways</title>
		<description><![CDATA[This September, RSRT brought together 150 leaders across biopharma and academia for the 2025 Rett Syndrome Genetic Medicines Summit. Over three days, the field aligned around one shared goal: advancing safe, effective genetic medicines that target the root cause of Rett syndrome.

The Summit created a focused environment for open, practical dialogue—where drug developers assessed the current landscape, scientists shared emerging insights, clinicians outlined real-world needs, and regulators clarified the path for advancing Rett programs toward the clinic. It underscored RSRT’s role as both curator and catalyst, helping translate promising ideas into meaningful progress.

Program Highlights

Speakers delivered clear, actionable updates across the genetic medicines landscape, offering a comprehensive view of the opportunities and challenges shaping Rett therapies. Poster sessions showcased new data from emerging scientists and industry teams, reflecting the breadth of innovation underway.]]></description>
		<pubDate>Tue, 18 Nov 2025 13:59:29 -0600</pubDate>
		<link>https://reverserett.org/news/articles/2025-rett-genetic-medicines-summit-highlights-next-steps/</link>
		<guid>https://reverserett.org/news/articles/2025-rett-genetic-medicines-summit-highlights-next-steps/</guid>
	</item>
	<item>
		<title>AI Meets Rett: RSRT Funds $1M Collaboration with Profluent to Advance Gene-editing with Artificial Intelligence</title>
		<description><![CDATA[I’m excited to share with our community a promising development that has been in the works for quite some time — RSRT has made a $1 million award to fund a partnership with Profluent Bio to apply artificial intelligence (AI) to gene editing for Rett syndrome. The collaboration was further solidified at our recent Rett Syndrome Genetic Medicines Summit. It’s a prime example of why that meeting of scientists, biopharma leaders, clinicians and regulatory experts is so important. Among the most exciting discussions at the Summit was a visionary roadmap for accelerated development of base editing therapeutics, presented by gene editing pioneer, Fyodor Urnov. 

A faster path to therapy

With the high-profile success of the Baby KJ treatment and based on recent interactions between regulators and leaders in the gene editing field including himself and fellow summit speaker David Liu, Fyodor outlined a novel platform-]]></description>
		<pubDate>Fri, 07 Nov 2025 12:00:21 -0600</pubDate>
		<link>https://reverserett.org/news/articles/profluent-rsrt-ai/</link>
		<guid>https://reverserett.org/news/articles/profluent-rsrt-ai/</guid>
	</item>
	<item>
		<title>Profluent and RSRT Partner to Target Rett Syndrome with AI-Designed Custom Genomic Medicines</title>
		<description><![CDATA[EMERYVILLE, Calif. – Profluent Bio announced a research collaboration with the Rett Syndrome Research Trust (RSRT) to design novel base editors to permanently correct mutations that cause Rett syndrome, a rare neurodevelopmental disorder. The partnership is grounded in a shared recognition that patients are waiting. Both organizations are committed to moving forward with urgency, rigor, and transparency.


“This collaboration is about people first – Rett families are trusting us with a challenge that affects them 24/7,” said Hilary Eaton, Chief Business Officer of Profluent. “There’s always been a tension between addressing rare diseases and making the economics work.  Profluent is committed to using AI as the equalizer and democratizing the field of gene editing; whether a patient has a rare or common disease, they deserve access to safe, high-quality treatments.” 


Rett syndrome is caused by mutations in the MECP2 gene. In this collaboration,]]></description>
		<pubDate>Fri, 14 Nov 2025 13:22:25 -0600</pubDate>
		<link>https://reverserett.org/news/press-releases/profluent-and-rsrt-partner-to-target-rett-syndrome-with-ai-designed-custom-genomic-medicines/</link>
		<guid>https://reverserett.org/news/press-releases/profluent-and-rsrt-partner-to-target-rett-syndrome-with-ai-designed-custom-genomic-medicines/</guid>
	</item>
	<item>
		<title>Neurogene Doses First Participant in Embolden™ Registrational Trial of NGN-401 for Rett Syndrome</title>
		<description><![CDATA[Neurogene announced today that the first participant has been dosed in their registrational clinical trial evaluating NGN-401, a gene therapy for Rett syndrome.

Key details:


	Single-arm, open-label, study in 20 females aged three years and older.
	Enrollment expected to complete within three to six months at 13 US clinical sites
	Trial intended to support a Biologics License Application (BLA) — a formal request to the FDA to approve a biological product, such as a gene therapy, for clinical use.
	Community letter to come shortly
]]></description>
		<pubDate>Thu, 06 Nov 2025 11:18:50 -0600</pubDate>
		<link>https://reverserett.org/news/articles/neurogene-doses-first-participant-in-embolden-trial-of-ngn-401-for-rett/</link>
		<guid>https://reverserett.org/news/articles/neurogene-doses-first-participant-in-embolden-trial-of-ngn-401-for-rett/</guid>
	</item>
	<item>
		<title>Taysha Receives FDA Breakthrough Therapy Designation for TSHA-102</title>
		<description><![CDATA[Taysha Gene Therapies has provided an update on its Rett gene therapy program via a press release and community letter. See links below.

Highlights include:


	
	FDA has granted TSHA-102 Breakthrough Therapy Designation, which expedites the development and review of treatments and dugs, based on preliminary safety and efficacy results from the first 12 participants in the REVEAL Phase 1/2 trial.
	
	
	Taysha has reached agreement with the FDA on the design of the REVEAL Pivotal Study, which will begin enrolling participants in late 2025. This study will include a 6-month interim analysis to evaluate whether participants gain or regain key developmental milestones.
	
	
	The pivotal trial will enroll 15 girls and young women between the ages of 6 to 22 with typical Rett syndrome. Enrollment is expected to begin this quarter.
	
	
	A separate study for younger girls (ages 2–6) is also planned.
	
	
	Taysha is exploring trial options outside the U.S.]]></description>
		<pubDate>Thu, 02 Oct 2025 10:28:50 -0500</pubDate>
		<link>https://reverserett.org/news/articles/taysha-receives-fda-breakthrough-therapy-designation-for-tsha-102/</link>
		<guid>https://reverserett.org/news/articles/taysha-receives-fda-breakthrough-therapy-designation-for-tsha-102/</guid>
	</item>
	<item>
		<title>Hey Universe! Science + Serendipity at RSRT</title>
		<description><![CDATA[This week we announced the expansion of our research team. And it struck me how once again RSRT’s growth has happened organically. Time and time again with each new hire the right person with the right qualifications has appeared. It’s really quite extraordinary.

 



 

Tim Freeman

The first major hire came in 2013. Like so many times before and since, I received a call from a mom whose 4-year-old daughter had been recently diagnosed with Rett. We talked about the usual stuff – therapies, schooling, research and at the end of our conversation she mentioned that her husband, Tim Freeman, worked in fundraising for a non-profit focused on education. Say no more. Tim and I were on the phone a week later and met in person shortly after that and the rest is history. Tim has been with RSRT for 12 years working closely with Rett families in the US and around the world who take action and fundraise for our genetic medicine programs.

 




Randy Carpenter, MD

The next major hire came in 2016,]]></description>
		<pubDate>Mon, 22 Sep 2025 19:30:57 -0500</pubDate>
		<link>https://reverserett.org/news/articles/hey-universe-science-serendipity-at-rsrt/</link>
		<guid>https://reverserett.org/news/articles/hey-universe-science-serendipity-at-rsrt/</guid>
	</item>
	<item>
		<title>RSRT Expands Internal Team to Speed the Advancement of Genetic Medicine Cures for Rett Syndrome</title>
		<description><![CDATA[The Rett Syndrome Research Trust (RSRT) is excited to announce a significant expansion of its research team, accelerating the organization’s mission to find cures for Rett syndrome, a devastating neurological genetic disorder caused by mutations in the MECP2 gene. The new team members bring expertise that complements the prodigious knowledge and experience of RSRT’s existing internal science staff—Robert Deans, PhD, Randall Carpenter, MD, and Jana Von Hehn, PhD.

John Sinnamon, PhD, has joined as Associate Director of Preclinical Research. Sinnamon first connected with RSRT founder and CEO, Monica Coenraads, when he was a postdoctoral researcher at Oregon Health &#x00026; Science University (OHSU) in 2014.  At OHSU, Sinnamon was part of a team developing RNA editing technology in the lab of Gail Mandel, PhD.  Sinnamon was first author on a trio of papers that put RNA editing on the map as a potential therapeutic strategy for Rett syndrome.]]></description>
		<pubDate>Fri, 26 Sep 2025 21:11:25 -0500</pubDate>
		<link>https://reverserett.org/news/press-releases/rsrt-expands-internal-team-to-speed-genetic-medicine-cures/</link>
		<guid>https://reverserett.org/news/press-releases/rsrt-expands-internal-team-to-speed-genetic-medicine-cures/</guid>
	</item>
	<item>
		<title>Taysha Advances REVEAL Pivotal Trial for Rett Syndrome</title>
		<description><![CDATA[Taysha announced today that their REVEAL pivotal trial is expected to begin enrollment in the last quarter of this year.  A pivotal clinical trial gathers a comprehensive set of data for review by regulatory agencies like the FDA or EMA with the goal of obtaining regulatory approval to market the therapeutic.

The study, which is open label, will enroll 15 girls and young women between the ages of 6 and under 22 years old. The primary endpoint in the study will measure the percentage of participants who gain or regain a least one milestone.

A separate safety study in girls between the ages of 2 and under 6 years is being planned.

Questions can be directed to patientaffairs@tayshagtx.com

More information is available by reading Taysha’s community letter and press release below.]]></description>
		<pubDate>Tue, 12 Aug 2025 08:37:32 -0500</pubDate>
		<link>https://reverserett.org/news/articles/taysha-advances-reveal-pivotal-trial-for-rett-syndrome/</link>
		<guid>https://reverserett.org/news/articles/taysha-advances-reveal-pivotal-trial-for-rett-syndrome/</guid>
	</item>
	<item>
		<title>Monica Coenraads Makes Forbes 50 Over 50 List</title>
		<description><![CDATA[We’re proud to share that our founder and CEO, Monica Coenraads, has been named to the 2025 Forbes 50 Over 50 list in the Impact category—one of four designations that celebrate women driving meaningful change across business, science, politics, and culture.

This annual list recognizes 50 women over the age of 50 who are using their insight and influence to leave the world better than they found it. Monica appears in remarkable company alongside visionaries like Maria Shriver, Halle Berry, and biotech leaders including Yvonne Greenstreet (CEO, Alnylam Pharmaceuticals), Jane Grogan (Head of Research, Biogen), and Vicky Sato (Chair, Vir Biotechnology).

As Forbes notes, Monica’s leadership began in deeply personal territory. When her daughter, Chelsea, was diagnosed with Rett in 1998, she transformed heartbreak into action. Within a year, she co-founded the first organization devoted to advancing Rett research, with a clear and unwavering goal: cure the root cause. Since then,]]></description>
		<pubDate>Fri, 01 Aug 2025 12:41:07 -0500</pubDate>
		<link>https://reverserett.org/news/articles/monica-honored-in-forbes-50-over-50/</link>
		<guid>https://reverserett.org/news/articles/monica-honored-in-forbes-50-over-50/</guid>
	</item>
	<item>
		<title>Neurogene Announces Trial Design for Embolden™ Study of NGN-401 Gene Therapy for Rett Syndrome</title>
		<description><![CDATA[Neurogene has announced key design elements of its upcoming registrational clinical trial, Embolden™, for NGN-401, an investigational gene therapy for Rett syndrome. A registrational clinical trial gathers a comprehensive set of data for review by regulatory agencies like the FDA or EMA with the goal of obtaining regulatory approval to market the therapeutic. 

Here are some highlights:


	The trial will include females aged 3 and older with a total of 18 patients expected to be enrolled.
	Participants will receive a single dose of NGN-401 in an open-label, single-arm, baseline-controlled study.
	The primary efficacy endpoint will assess improvement over a 12 month period using the CGI-Improvement scale, along with the gain of at least one developmental milestone or skill from a list of 28.


Trial site locations have not yet been determined, and enrollment is not yet open.]]></description>
		<pubDate>Fri, 27 Jun 2025 18:38:49 -0500</pubDate>
		<link>https://reverserett.org/news/articles/neurogene-embolden-announce-june30-2025/</link>
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