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FAQ on GeneTx Acquisition

What is being announced?

On July 18, 2022, FAST announced that the company Ultragenyx Pharmaceuticals had exercised its option and closed on its acquisition of GeneTx Biotherapeutics LLC, a biotechnology company founded by FAST. 

Until this month, FAST maintained a 58% majority ownership stake in the company. With this latest announcement, GeneTx will be folded into Ultragenyx, which will assume complete control of the GTX-102 program, including the ongoing clinical trial.  

What is GeneTx, and why did FAST start it?

GeneTx is a for-profit company founded specifically for one purpose: to accelerate the development of an investigational antisense oligonucleotide drug, GTX-102, for the potential treatment of AS. It was launched as one of multiple strategies under FAST’s signature “C.U.R.E.-AS” approach. 

A bit of history is useful here: FAST was founded in 2008 by incredible visionaries, led by Paula Evans and a group of highly motivated parents, who were driven by a singular mission: to cure Angelman syndrome. This vision laid the groundwork for incredible progress. In 2013, The FAST FIRE team, led by Dr. Ed Weeber, in collaboration with Dr. Scott Dindot, Kevin Nash, David Segal and Anne Anderson, created one of the first truly collaborative research efforts to advance the mission of FAST, driving different concepts and modalities forward for potential human application. Then, in 2015, Dr. Allyson Berent joined the board of directors at FAST, and in 2016 became the Chief Science Officer. With her focus and expertise in translational research, as a clinical researcher herself, she was able to help FAST drive the development of an innovative “Roadmap to a Cure”—an effort to bring together lead scientists from around the world to further their technology to move from benchside to bedside. 

One of the early insights, which began to drive all of FAST’s work, was that successful treatments are almost never discovered on their own; the more efforts underway in a given field—and the more diverse—the more likely it would be to find truly effective therapeutics.

Drug development process illustration for the GTX FAQ page

In other words: To get to one meaningful treatment, we needed to start with a field of thousands of initial options. 

To this end, early funding from the Angelman community went to support dozens of scientific heroes—whose work was, and continues to be, the foundation for so much of the early progress made in the field. It has been an incredible team that is working together vigorously to meet the mission of FAST.

Inspired by the ambition and possibility of FAST’s roadmap and the FIRE team, the Marnier Lapostalle Foundation donated $5.8m in 2016—the largest ever fundraising gift for Angelman syndrome—which  six disease modifying strategies to get from proof-of-concept in cell lines and animal models to human drug candidates, with the goal to have therapies that could reach human clinical trials. 

One of those early grants went to Dr. Scott Dindot of Texas A&M University, who was already being funded by FAST to further uncover the genetics behind the imprinting phenomenon in Angelman syndrome which is responsible for the unique silencing of the father’s copy of the UBE3A gene in neurons. In the wake of this new gift, Dr. Dindot discovered a unique region on the UBE3A-ATS transcript, which had important implications in unsilencing, and therefore turning on, the UBE3A gene on the paternal copy.  

Motivated by this important work, later that same year FAST created a for-profit company called GeneTx Biotherapeutics LLC specifically for one purpose: to accelerate the development of an investigational antisense oligonucleotide drug for the potential treatment of AS.GeneTx soon attracted the attention of, and later investment byUltragenyx Pharmaceuticals—a company focused on rare diseases, with a reputation for being uncommonly patient-centered. With an investment by Ultragenyx of $25m, GeneTx was able to advance GTX-102 into a clinical trial.

In February of 2020, the clinical trial was initiated, making this FAST-backed program the first potential disease-modifying therapy in human clinical trials for Angelman syndrome. 

How does an ASO work and how might it help my loved one with AS?

This video explains the concept of how an antisense oligonucleotide could activate the silent paternal (father’s) copy of the gene in neurons of the brain to give back a functional copy of the UBE3A gene to individuals living with Angelman syndrome.

So, what’s happening now?

As noted, GeneTx and Ultragenyx have been working together to conduct a Phase 1/2 clinical study of GTX-102 in the U.S., Canada and the United Kingdom. 

In July, Ultragenyx exercised its exclusive option and closed on the acquisition of GeneTx, and will now take over the GTX-102 clinical program. We are incredibly optimistic that this program in Ultragenyx hands will further advance development of this potential therapeutic throughout the world.

When can we expect to hear the results from those trials?

Ultragenyx issued a press release which included promising interim data from the Phase 1/2 study.

What can people with AS and their families expect in the coming weeks and months as a result of this sale?

We know you have questions. What we feel confident saying is that Ultragenyx exercising its option to buy GeneTx would seem to suggest that the company sees great potential for this program.

Will this have any impact on the current clinical studies of the investigational antisense oligonucleotide drug, GTX-102, for the potential treatment of AS?

FAST is not involved with running the clinical trial, so we cannot comment on study conduct. We can say GeneTx sought Ultragenyx out as its partner for clinical development of GTX-102 in 2019 because Ultragenyx has the right expertise and capabilities to ensure a robust clinical program and regulatory strategy for GTX-102 is executed upon.

What does this sale mean for FAST, and the AS community as a whole?

What happened today is why FAST was founded, why we started GeneTx, and why we donate and fundraise: To discover and accelerate promising therapies for AS.

Ultragenyx is a biopharmaceutical company involved in the research and development of novel products for the treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and a high unmet medical need. The company has the resources and experience needed to push the development of GTX-102 forward with the upmost care. Having Ultragenyx in the driver’s seat for the ongoing clinical trial and program lifecycle makes us very proud, and confident that it is in the best of hands. 

But the benefits go far beyond that, as FAST itself is now able to pledge millions of dollars to be used directly to supercharge numerous pathways and promising programs, and in the process enrich the entire landscape of AS-related therapeutic research. Over the past few months, numerous programs in biotech have been paused, shelved, or cancelled due re-prioritization of programs and tumultuous financial markets—a trend that could derail potentially promising Angelman programs from being advanced. With FAST now able to focus on acceleration and progress, we can lend support to help advance a host of promising opportunities.

Why should I continue to raise funds for FAST now that the organization has received so much money from this transaction?

Because we aren’t there yet! The platforms currently being pursued and advanced through donations to FAST include AAV gene therapy, hematopoietic stem cell gene therapy, enzyme replacement therapy, CRISPR technology, miRNA, and many more. FAST has not stopped pushing the timelines to help these programs get closer to human clinical trials in order to ensure that no stone is left unturned. You can see what we mean here:

Graphic: Angelman syndrome therapeutic pipeline

When we look at a budget to get to Phase 1/2 clinical trials for all of these promising programs, we believe we need to raise at least $200M. The proceeds from the sale of GeneTx shares, which will be carefully invested, get us much closer than we ever imagined possible at this point—putting wind at our backs, at a crucial moment. A number of FAST-funded programs are already showing considerable promise in animal models. If we want to help ensure that there are meaningful options for our loved ones, we must drive each program as far as we can—a goal that we believe can be met through supporting existing company programs, advancing our own academic programs, and ensuring we find the best partnerships, like we did with Ultragenyx, when the time is right.

Following the transaction, will anyone from FAST be involved in Ultragenyx’s GTX-102 program?

Dr. Allyson Berent, Chief Science Officer at FAST, has been working on the development program for GTX-102 since 2017 and will serve as a consultant for Ultragenyx.

Are any of the GeneTx team members who are part of FAST benefiting financially from the transaction?

Yes. We cannot discuss specific terms or details of this acquisition. But as with all such transactions, individuals who invested time or money to make the initial operation a reality will likely receive returns on those outlays. In fact, we wish we could name them—and thank them publicly—since without their generosity, faith and willingness to take a risk for the possibility of a treatment, we wouldn’t be where we are today.

Is FAST going to be biased toward GTX-102 over other pharmaceutical companies’ therapeutics?

No. Remember: Every single board member of FAST is a parent to a child living with Angelman syndrome! We are true to our mission. FAST’s time and money have gone and will continue to go to supporting all companies and all shots on goal to ultimately cure Angelman syndrome. FAST has continued to support all kinds of promising research—from AAV gene therapy to hematopoietic stem cell gene therapy, to enzyme replacement therapy and other gene editing and down-stream targets. In addition, FAST has invested millions of dollars into initiatives to support all pharmaceutical companies, whether academic sponsored or industry sponsored. FAST has led by example in a collaborative spirit since the inception of GeneTx in 2017, and has the intention of doing far more now to support all stakeholders with this infusion of funds.

How do you think other pharmaceutical companies will react to this news? What about scientists and those in the medical community?

Our top priority as an advocacy organization is finding meaningful treatment options for AS, and we share that goal with many stakeholders across the pharmaceutical industry, research institutions, and of course the Angelman syndrome community. As before, we will support everyone and anyone with a chance to make that happen.

As we have been saying all along: #BetterDaysAre Coming. Join us in our effort to get there—FAST! 

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