Baylor center receives $10M NIH grant to continue rare disease research

NIH funding

BCM's Center for Precision Medicine Models has received funding that will allow it to study more complex diseases. Photo via Getty Images

Baylor College of Medicine’s Center for Precision Medicine Models received a $10 million, five-year grant from the National Institutes of Health last month that will allow it to continue its work studying rare genetic diseases.

The Center for Precision Medicine Models creates customized cell, fly and mouse models that mimic specific genetic variations found in patients, helping scientists to better understand how genetic changes cause disease and explore potential treatments.

The center was originally funded by an NIH grant, and its models have contributed to the discovery of several new rare disease genes and new symptoms caused by known disease genes. It hosts an online portal that allows physicians, families and advocacy groups to nominate genetic variants or rare diseases that need further investigation or new treatments.

Since its founding in 2020, it has received 156 disease/variant nominations, accepted 63 for modeling and produced more than 200 precision models, according to Baylor.

The center plans to use the latest round of funding to bring together more experts in rare disease research, animal modeling and bioinformatics, and to expand its focus and model more complex diseases.

Dr. Jason Heaney, associate professor in the Department of Molecular and Human Genetics at BCM, serves as the lead principal investigator of the center.

“The Department of Molecular and Human Genetics is uniquely equipped to bring together the diverse expertise needed to connect clinical human genetics, animal research and advanced bioinformatics tools,” Heaney added in the release. “This integration allows us to drive personalized medicine forward using precision animal models and to turn those discoveries into better care for patients.”

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A new AI tool from a Baylor College of Medicine Lab could help better diagnose specific types of autism spectrum disorder, epilepsy and developmental delay disorders. Photo via Getty Images.

Houston lab develops AI tool to improve neurodevelopmental diagnoses

developing news

One of the hardest parts of any medical condition is waiting for answers. Speeding up an accurate diagnosis can be a doctor’s greatest mercy to a family. A team at Baylor College of Medicine has created technology that may do exactly that.

Led by Dr. Ryan S. Dhindsa, assistant professor of pathology and immunology at Baylor and principal investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, the scientists have developed an artificial intelligence-based approach that will help doctors to identify genes tied to neurodevelopmental disorders. Their research was recently published the American Journal of Human Genetics.

According to its website, Dhindsa Lab uses “human genomics, human stem cell models, and computational biology to advance precision medicine.” The diagnoses that stem from the new computational tool could include specific types of autism spectrum disorder, epilepsy and developmental delay, disorders that often don’t come with a genetic diagnosis.

“Although researchers have made major strides identifying different genes associated with neurodevelopmental disorders, many patients with these conditions still do not receive a genetic diagnosis, indicating that there are many more genes waiting to be discovered,” Dhindsa said in a news release.

Typically, scientists must sequence the genes of many people with a diagnosis, as well as people not affected by the disorder, to find new genes associated with a particular disease or disorder. That takes time, money, and a little bit of luck. AI minimizes the need for all three, explains Dhindsa: “We used AI to find patterns among genes already linked to neurodevelopmental diseases and predict additional genes that might also be involved in these disorders.”

The models, made using patterns expressed at the single-cell level, are augmented with north of 300 additional biological features, including data on how intolerant genes are to mutations, whether they interact with other known disease-associated genes, and their functional roles in different biological pathways.

Dhindsa says that these models have exceptionally high predictive value.

“Top-ranked genes were up to two-fold or six-fold, depending on the mode of inheritance, more enriched for high-confidence neurodevelopmental disorder risk genes compared to genic intolerance metrics alone,” he said in the release. “Additionally, some top-ranking genes were 45 to 500 times more likely to be supported by the literature than lower-ranking genes.”

That means that the models may actually validate genes that haven’t yet been proven to be involved in neurodevelopmental conditions. Gene discovery done with the help of AI could possibly become the new normal for families seeking answers beyond umbrella terms like “autism spectrum disorder.”

“We hope that our models will accelerate gene discovery and patient diagnoses, and future studies will assess this possibility,” Dhindsa added.

Baylor Genetics has paired with Baylor’s department of molecular and human genetics to launch the Medical Genetics Multiomics Laboratory with a goal for the collaboration is to turn research into clinical diagnostics. Photo via Getty Images

This new Houston lab is translating genetics research into clinical diagnostics

DNA innovation

A new lab at Baylor College of Medicine is primed to do groundbreaking work in the field of genetics.

Baylor Genetics has paired with Baylor’s department of molecular and human genetics to launch the Medical Genetics Multiomics Laboratory (MGML). The goal for the collaboration is to turn research into clinical diagnostics.

MGML’s freshly launched first clinical test is Whole Transcriptomic RNA Sequencing (WT RNAseq). The new test builds upon the success of existing tests like whole exome sequencing (WES) and whole genome sequencing (WGS) currently on offer from Baylor Genetics by focusing on additional variants that could be missed by the other tests.

Baylor Genetics is offering WT RNAseq to the Undiagnosed Diseases Network (UDN) and its affiliated institutions. For more than a decade, the NIH-funded UDN has united clinical and research experts from across many fields and institutions to give answers to patients with rare genetic diseases. Since it became one of the first institutions to join the UDN in 2014, Baylor Genetics has been the UDN’s sequencing core, using WES, WGS and RNA sequencing to help diagnose patients. The additional offering of WT RNAseq could improve the diagnostic yield by as much as 17 percent.

“This agreement, and the MGML lab, bring to life our vision of innovation, allowing us to co-develop new tests, evaluate in terms of clinical utility, and offer commercially in either a research or clinical setting,” says Dr. Brendan Lee, professor, chair and Robert and Janice McNair Endowed Chair of Molecular and Human Genetics at Baylor College of Medicine, and scientific advisory and board of directors member at Baylor Genetics. “Baylor Genetics is turning around critical high-volume testing, but the challenge is also maintaining our innovative edge and our position as leaders in discovery and genomic health implementation. This agreement is a realization of the vision when Baylor Genetics was founded 10 years ago.”

The lab’s product offerings will continue to expand as it becomes commercially feasible to do so, and the new tests will be used both commercially and clinically.

Baylor Genetics combines the powers of Baylor College of Medicine, which has the NIH’s best-funded department of molecular and human genetics, and Japanese clinical diagnostic testing company H.U. Group Holdings.

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CPRIT CEO: Houston’s $2B in funding is transforming cancer research and prevention

fighting cancer

With its plethora of prestigious health care organizations like the University of Texas MD Anderson Cancer Center, UTHealth Houston, and the Baylor College of Medicine, Houston sits at the heart of cancer research and prevention in Texas.

Of course, it takes piles of cash to support Houston’s status as the state’s hub for cancer research and prevention. Much of that money comes from the Cancer Prevention and Research Institute of Texas (CPRIT).

Data supplied by CPRIT shows organizations in Harris County gained $2.3 billion in institute funding from 2009 through 2025, or nearly $145 million per year. That represents almost 60 percent of the roughly $4 billion that CPRIT has granted to Texas institutions over a 16-year period.

“The life sciences ecosystem that has developed and changed in Houston is phenomenal,” Kristen Doyle, who became the agency’s CEO in July 2024, tells InnovationMap. “In the next decade, we will look back and see a great transformation.”

That ecosystem includes more than 1,100 life sciences and biotech companies, according to the Greater Houston Partnership.

Houston plays critical role in clinical trials

Texas voters approved the creation of CPRIT in 2007. Twelve years later, voters agreed to earmark an extra $3 billion for CPRIT, bringing the state agency’s total investment in cancer research and prevention to $6 billion.

To date, CPRIT money has gone toward recruiting 344 cancer researchers to Texas (mainly to Houston) and has supported cancer prevention services for millions of Texans in the state’s 254 counties. CPRIT funding has also helped establish, expand, or relocate 25 cancer-focused companies. In Houston, MD Anderson ranks as the No. 1 recipient of CPRIT funding.

Regarding cancer research, Doyle says Houston plays a critical role in clinical trials.

“[Clinical trials are] something that CPRIT has focused on more and more. Brilliant discoveries are crucial to this whole equation of solving the cancer problem,” Doyle says. “But if those brilliant ideas stay in the labs, then we’ve all failed.”

Researchers conduct more clinical trials in Houston than anywhere else in the U.S., the Greater Houston Partnership says.

Doyle, a 20-year survivor of leukemia, notes that a minority of eligible patients participate in clinical trials for cancer treatments, “and that’s one of the reasons that it takes so long to get a promising drug to market.”

An estimated 7 percent of cancer patients sign up for clinical trials, according to a study published in 2024 in the Journal of Clinical Oncology.

MD Anderson takes on cancer prevention

Doyle also notes that Houston is leading the charge in cancer prevention.

“We get some national recognition for programs that have been developed in Houston that then can be replicated in other parts of the country,” she says.

Much of the work in Houston focusing on cancer prevention takes place at MD Anderson. The hospital reports that it has received more than $725 million from the CPRIT since 2007, representing approximately 18 percent of CPRIT’s total awards.

“These efforts can have profound impact on the lives of patients and their families, and this funding ensures our exemplary clinicians and scientists can continue working together to drive breakthroughs that advance our mission to end cancer,” Dr. Giulio Draetta, chief scientific officer at MD Anderson, said in a November news release, following the most recent CPRIT award for the hospital totalling more than $29 million.

CPRIT funding for Houston institutions supplements the more than $4.5 billion in federal funding for health and life sciences research and innovations that the Houston area received from 2020 to 2024, according to the Greater Houston Partnership.

“We are curing cancer every single day,” Doyle says of CPRIT. “Every step that we are taking — whether that’s funding great ideas or funding the clinical trials that are bringing promising drugs to Texas and to the world — we are making a difference.”

Houston energy tech co. breaks ground on low-cost hydrogen pilot plant

Coming Soon

Houston’s Lummus Technology and Advanced Ionics have broken ground on their hydrogen pilot plant at Lummus’ R&D facility in Pasadena, Texas.

The plant will support Advanced Ionics’ cutting-edge electrolyzer technology, which aims to deliver high-efficiency hydrogen production with reduced energy requirements.

“By demonstrating Advanced Ionics’ technology at our state-of-the-art R&D facility, we are leveraging the expertise of our scientists and R&D team, plus our proven track record of developing breakthrough technologies,” Leon de Bruyn, president and CEO of Lummus, said in a news release. “This will help us accelerate commercialization of the technology and deliver scalable, cost-effective and sustainable green hydrogen solutions to our customers.”

Advanced Ionics is a Milwaukee-based low-cost green hydrogen technology provider. Its electrolyzer converts process and waste heat into green hydrogen for less than a dollar per kilogram, according to the company. The platform's users include industrial hydrogen producers looking to optimize sustainability at an affordable cost.

Lummus, a global energy technology company, will operate the Advanced Ionics electrolyzer and manage the balance of plant systems.

In 2024, Lummus and Advanced Ionics established their partnership to help advance the production of cost-effective and sustainable hydrogen technology. Lummus Venture Capital also invested an undisclosed amount into Advanced Ionics at the time.

“Our collaboration with Lummus demonstrates the power of partnerships in driving the energy transition forward,” Ignacio Bincaz, CEO of Advanced Ionics, added in the news release. “Lummus serves as a launchpad for technologies like ours, enabling us to validate performance and integration under real-world conditions. This milestone proves that green hydrogen can be practical and economically viable, and it marks another key step toward commercial deployment.”

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This article originally appeared on EnergyCapitalHTX.com.

TMC launches new biotech partnership with Republic of Korea

international collaboration

Houston's Texas Medical Center has launched its new TMC Republic of Korea BioBridge.

The new partnership brings together the TMC with the Osong Medical Innovation Foundation, or KBIOHealth. The Biobridge aims to support the commercialization of Korean biotech and life science startups in the U.S., foster clinical research, and boost collaboration in the public, private and academic sectors.

Through the partnership, TMC will also develop a Global Innovators Launch Pad to foster U.S. market entry for international health care companies. Founders will be selected to participate in the 10-week program at the TMC Innovation Factory in Houston.

“Gene and cell therapies are driving biotech innovation, opening possibilities for treating diseases once thought untreatable," William McKeon, president and CEO of the Texas Medical Center, said in a news release. "Expanding biomanufacturing capacity is essential to delivering the next wave of these therapies, and partnerships with leading innovators will strengthen our efforts in Houston and internationally.”

McKeon officially signed the TMC Korea BioBridge Memorandum of Understanding with Myoung Su Lee, chairman of KBIOHealth, in South Korea in October.

"This collaboration marks a significant milestone for Korea’s biohealth ecosystem, creating a powerful bridge between Osong and Houston," Lee added in the release. "By combining KBIOHealth’s strength in research infrastructure and Korea’s biotech talent with TMC’s global network and accelerator platform, we aim to accelerate innovation and bring transformative solutions to patients worldwide.”

This is the seventh international strategic partnership for the TMC. It launched its first BioBridge with the Health Informatics Society of Australia in 2016. It launched its TMC Japan BioBridge, focused on advancing cancer treatments, last year. It also has BioBridge partnerships with the Netherlands, Ireland, Denmark and the United Kingdom.

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