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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UH lands $11.8M for first-of-its-kind early language development study

speech funding

Researchers at the University of Houston have secured an $11.8 million grant from the National Institutes of Health to conduct a first-of-its-kind study of early language development.

Led by Elena Grigorenko, the Hugh Roy and Lillie Cranz Cullen Distinguished Professor of Psychology, and research professor Jack Fletcher, the study will follow 3,600 children aged 18 to 24 months to uncover how language skills develop at this critical stage and why some children experience delays that can influence later growth.

The NIH funding will also support the development of the new national Clinical Research Center on Developmental Language Disorders at UH, which aims to bring experts from psychology, education, health and measurement sciences to study how children learn language.

“This will be the first national study to estimate how common late talking is using a large, representative sample of Houston toddlers,” Grigorenko said in a news release. “By following these children as they grow, we hope to better understand the developmental pathways that can lead to conditions such as developmental language disorder and autism.”

UH’s team will partner with the pediatric clinic network at Texas Children’s Hospital, where children will be screened for early language development, allowing researchers to identify those who show signs of delayed speech. Next, researchers will follow the cohort through early childhood to examine how language abilities evolve and how early delays may lead to later challenges.

The Clinical Research Center on Developmental Language Disorders will be the 14th national research center established at UH, and will include researchers from multiple UH departments, as well as partners at Baylor College of Medicine and the Texas Center for Learning Disorders.

“This level of investment from the National Institutes of Health reflects the significance of this work to address a complex challenge affecting children, families and communities,” Claudia Neuhauser, vice president for research at UH, said in a news release. “By bringing together experts from multiple disciplines and partnering with major health systems across the region, the project reflects our commitment to advancing discoveries that impact our community.”

Rice Alliance names Houston healthtech exec as first head of platform

new hire

The Rice Alliance for Technology and Entrepreneurship has named its first head of platform.

Houston entrepreneur Laura Neder stepped into the newly created role last month, according to an email from Rice Alliance. Neder will focus on building and growing Houston’s Venture Advantage Platform.

The emerging platform, which is being promoted by Rice Alliance and the Ion, aims to connect founders with the "people, capital and expertise they need to scale."

"I’ve spent a lot of time thinking about what it takes to make an innovation ecosystem more navigable, more connected, and more useful for founders," Neder said in a LinkedIn post. "I’m grateful for the opportunity to do that work at Rice Alliance, alongside a team with a long history of supporting entrepreneurship and innovation."

"Houston has the talent, institutions, and industry base to create real advantage for founders," she added. "I’m looking forward to listening, learning, and building stronger pathways across the ecosystem."

Neder most recently served as CEO of Houston-based Careset, where she helped bring the Medicare data startup to commercialization. Prior to that, Neder served as COO of Houston-based telemedicine startup 2nd.MD, which was acquired for $460 million by Accolade in 2021.

"Laura brings a rare combination of founder empathy, operational experience and ecosystem leadership," Rice Alliance shared.

Neder and Rice Alliance also shared that the organization is hiring developers to design the new Venture Advantage Platform. Learn more here.

Elon Musk's SpaceX files initial paperwork to sell shares to the public

Incoming IPO

Elon Musk's space exploration company has filed preliminary paperwork to sell shares to the public, according to two sources familiar with the filing, a blockbuster offering that would likely rank as the biggest ever and could make its founder the world's first trillionaire.

A SpaceX IPO promises to be one of the biggest Wall Street events of the year, with several investment banks lining up to help raise tens of billions to fund Musk's ambitions to set up a base on the moon, put datacenters the size of several football fields in orbit and possibly one day send a man to Mars.

The sources spoke on condition of anonymity because they were not authorized to talk publicly about the confidential registration with the Securities and Exchange Commission.

SpaceX did not respond immediately to a request for comment.

Exactly how much SpaceX plans to raise has not been disclosed but the figure is reportedly as much as $75 billion. At that level, the offering would easily eclipse the $29 billion that Saudi Aramco raised in its IPO in 2019.

The offering, coming possibly in June, could value all the shares of SpaceX at $1.5 trillion, nearly double what the company was valued in December when some minority owners sold their stakes, according to research firm Pitchbook, before an acquisition that increased its size.

Musk owns 42% of the SpaceX now, according to Pitchbook, though that figure will change with the IPO when new owners are issued shares. In any case, he is likely to pierce the trillion dollar mark because he is already close. Forbes magazine estimates Musk's net worth at roughly $823 billion.

In addition to making reusable rockets to hurl astronauts and hardware into orbit, SpaceX owns Starlink, the world’s largest satellite communications company. The company also recently brought under its roof two other Musk businesses, social media platform X, formerly Twitter, and artificial intelligence business, xAI, in a controversial transaction because both the seller and the buyer were controlled by him.

SpaceX has become the biggest commercial launch company in its industry, responsible for sending payloads into orbit for customers across the globe, but has also benefited from big taxpayer spending. That has raised conflicts of interest issues given that Musk was the biggest donor to President Donald Trump's campaign and is still a big backer.

In the past five years, SpaceX won $6 billion in contracts from NASA, the Defense Department and other U.S. government agencies, according to USAspending.gov.

Among current SpaceX owners is Donald Trump Jr, the president's oldest son. He owns a shares through 1789 Capital. That venture capital firm made him a partner shortly after his father won the presidency for a second time and has been buying up federal contractors seeking to win taxpayer money ever since.

The White House and Trump himself have repeatedly denied there are any conflicts of interest between his role as president and his family's businesses.

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