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Uniting for Progress – the Fifth Annual SYNGAP1 Conference hosted by SynGAP Research Fund (SRF) will take place November 30th in Orlando, Florida. #SyngapConf

Orlando, FL – 19 October 2023 – The Syngap Research Fund (SRF) will host Uniting for Progress — its fifth annual conference on SYNGAP1 research…

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Orlando, FL – 19 October 2023 – The Syngap Research Fund (SRF) will host Uniting for Progress — its fifth annual conference on SYNGAP1 research and clinical care on Thursday, November 30 at the Embassy Suites in Orlando, Florida.  Clinicians, researchers, industry professionals and SYNGAP1 families are invited to register at SyngapResearchFund.org.

Credit: SRF

Orlando, FL – 19 October 2023 – The Syngap Research Fund (SRF) will host Uniting for Progress — its fifth annual conference on SYNGAP1 research and clinical care on Thursday, November 30 at the Embassy Suites in Orlando, Florida.  Clinicians, researchers, industry professionals and SYNGAP1 families are invited to register at SyngapResearchFund.org.

“Uniting for Progress will showcase how SRF and the SYNGAP1 community are ready to partner with industry to deliver therapies for patients with this horrible disease.  It is an important opportunity for us to collaboratively improve the lives of people with SYNGAP1” said Mike Graglia, Managing Director of SRF. 

“The SRF annual scientific conference is an excellent opportunity to learn about the latest advances in SYNGAP1 research and to collaborate with other researchers on new projects. I am excited to be a part of this event and to help make a difference in the lives of people with SYNGAP1 Related Disorder,” said Dr. Kim Wiltrout, MD, of Boston Children’s Hospital.

The agenda will feature six sections:

  • New Findings about SYNGAP1
  • Drug Repurposing for SYNGAP1
  • Understanding SYNGAP1 at a Molecular Level – VUS & Missense Variants
  • Updates on Public Preclinical Pipelines
  • Clinical Trial Readiness – Natural History
  • Clinical Trial Readiness – Quantitative Measures

The scientific conference on Thursday will be followed by a family meeting on Friday, December 1, 2023 at the same location. Families are encouraged to attend both days.

“The conference is a pivotal annual event for the SYNGAP1 community. It is an invaluable opportunity to learn about the latest SYNGAP1 research, network with professionals who understand our children, bond with other families, and advocate for our loved ones. Coming together once a year fuels my passion and energy to be part of the SRF team building community and seeking precision therapies for our children,” said Suzanne Jones, parent of a child with SYNGAP1 & SRF Board Chair

We are grateful to our sponsors Stoke Therapeutics, Acadia Pharmaceuticals, Simons Searchlight, Tevard Biosciences, ciitizen/Invitae, Longboard Pharmaceuticals, and Rarebase for their support of this event.  

“Every year this event continues to grow – more families, more researchers, more clinicians – and we couldn’t do it without our sponsors,” said Peter Halliburton, Director of Development for SRF.

Agenda with topics and speakers

  • New Findings
    • New insights in the DEEs, including SYNGAP1 by Prof. Scheffer, AO, MBBS, PhD, University of Melbourne
    • Gene delivery by milk exosomes restores SYNGAP1 expression in mouse brains by Prof. Zempleni, PhD, University of Nebraska
    • SYNGAP1 beyond the Synapse by Dr. Willsey, PhD, University of California, San Francisco
       
  • Drug Repurposing
    • Moderation by Dr. Xin Tang, PhD, of Boston Children’s Hospital
    • Drug Repurposing Screen in Drosophila by Dr. Chow, PhD, University of Utah
    • Drug Repurposing Screen in Patient Models by Dr. Moxham, PhD, Rarebase, PBC
    • Lessons Learned from Phenylbutyrate Repurposing by Dr. Grinspan, MD, MS, Weill Cornell Medicine
       
  • Understanding SYNGAP1 at a Molecular Level
    • SynGAP missense: potential druggability and how might we get there by Dr. Courtney, PhD, Turku Bioscience Centre, Finland
    • Modeling the structural effects of SYNGAP1 missense mutations using molecular dynamics simulations by Dr. Postila, PhD, Turku Bioscience Centre, Finland
    • Integrated approaches to resolving SYNGAP1 missense variants of uncertain significance by Dr. Carville, PhD, Northwestern University
    • iPSC models of SYNGAP1 dysfunction by Dr. Coba, PhD, University of Southern California
    • Non-synaptic function of the ASD Associated Gene SYNGAP1 in Cortical Neurogenesis by Dr. Birtele, PhD, University of Southern California
       
  • Preclinical Pipeline
    • Why SYNGAP1 is an attractive target for Industry by Dr. Mingorance, PhD, Dracaena Consulting
    • TANGO Platform by Dr. Aznarez, PhD, Stoke Therapeutics
    • Praxis ASO Platform by Praxis Precisions Medicine
    • Suppressor tRNAs for the treatment of DEEs by Daniel Fisher, MBA, Tevard Biosciences
       
  • Clinical Trial Readiness – Natural History
    • Moderation by Dr. Helbig, MD, Children’s Hospital of Philadelphia 
    • A prospective natural history study in SYNGAP1 – first insights from ENDD by Dr. Jillian McKee, MD, Children’s Hospital of Philadelphia
    • Understanding the natural history of SYNGAP1 through data integration by Julie Xian, Children’s Hospital of Philadelphia
    • Outlining the clinical landscape of SYNGAP1 through a computational phenotype approach using 5586 phenotypic annotations in 197 individuals by Dr. Kessler, MD, Children’s Hospital of Philadelphia
    • SYNGAP1 Genotype and Phenotype Analysis by Dr. Kim Wiltrout, MD, Boston Children’s Hospital 
    • Meaningful Clinical Outcomes and Development of a Disease Concept Model by Katharine Cunnane, Weill Cornell Medicine
       
  • Clinical Trial Readiness – Quantitative Measures
    • Using EEG to understand “how the brain works” in SYNGAP1 by Dr. Levin, MD, Boston Children’s Hospital
    • Deep Learning EEG Biomarkers in SYNGAP1 Rodent Models and Patients by Dr. Gonzalez-Sulser, PhD, University of Edinburgh
    • Validating the ORCA for SYNGAP1 & other DEEs by Dr. Zigler, PhD, Duke University
    • Recent Neurobehavioral Findings in SYNGAP1 by Dr. Frazier, PhD, John Carroll University

About SYNGAP1-related intellectual disability (SRID)

SYNGAP1-related intellectual disability (ICD-10 F78.A1) is a rare genetic disorder caused by variants on the SYNGAP1 gene that reduce SynGAP protein levels. SRF has identified almost over 1,300 patients to date, the number grows weekly.  This protein acts as a regulator in the synapses (where neurons communicate with each other). When SynGAP protein levels are too low, we see an increase in excitability in the synapses making it difficult for neurons to communicate effectively. This leads to many neurological issues seen in SynGAP patients.

Symptoms of SYNGAP1 include: intellectual disability; epilepsy; hypotonia (low muscle tone); gross and fine motor skill delays; autism spectrum disorder; gastro-intestinal disorders; sleep and behavior disorders and visual abnormalities. 

About the SynGAP Research Fund (SRF)

The mission of the SynGAP Research Fund (SRF) is to improve the quality of life for SYNGAP1 patients through the research and development of treatments, therapies and support systems. 

SRF was founded in the US in 2018 as a 501(c)(3) US public charity, and families created sister organizations for SRF in the UK in 2020, in Europe (Netherlands) in 2022, and in Latin America (Colombia) in 2023. 

Completely parent-led, SRF is the largest non-government funder of SynGAP research having committed over $4 million in grants to date. The founders cover all operational costs, ensuring donations fund science. SRF’s grant program awards one or two-year grants to investigators, physician residents, and clinicians who are interested in studying SYNGAP1. SRF grants are intended to help researchers explore novel ideas and answer open questions related to the clinical aspects of and therapies for SRID. 

SRF is a member of the COMBINEDbrain, Global Genes Foundation Alliance, the Everylife Foundation Community Congress, Personalized Medicine Coalition, Rare Epilepsy Network, and the Epilepsy Leadership Council.

For more on SRF, visit: SyngapResearchFund.org or follow @cureSYNGAP1 on X, LinkedIn, YouTube, Instagram, Facebook or TikTok.


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Glimpse Of Sanity: Dartmouth Returns Standardized Testing For Admission After Failed Experiment

Glimpse Of Sanity: Dartmouth Returns Standardized Testing For Admission After Failed Experiment

In response to the virus pandemic and nationwide…

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Glimpse Of Sanity: Dartmouth Returns Standardized Testing For Admission After Failed Experiment

In response to the virus pandemic and nationwide Black Lives Matter riots in the summer of 2020, some elite colleges and universities shredded testing requirements for admission. Several years later, the test-optional admission has yet to produce the promising results for racial and class-based equity that many woke academic institutions wished.

The failure of test-optional admission policies has forced Dartmouth College to reinstate standardized test scores for admission starting next year. This should never have been eliminated, as merit will always prevail. 

"Nearly four years later, having studied the role of testing in our admissions process as well as its value as a predictor of student success at Dartmouth, we are removing the extended pause and reactivating the standardized testing requirement for undergraduate admission, effective with the Class of 2029," Dartmouth wrote in a press release Monday morning. 

"For Dartmouth, the evidence supporting our reactivation of a required testing policy is clear. Our bottom line is simple: we believe a standardized testing requirement will improve—not detract from—our ability to bring the most promising and diverse students to our campus," the elite college said. 

Who would've thought eliminating standardized tests for admission because a fringe minority said they were instruments of racism and a biased system was ever a good idea? 

Also, it doesn't take a rocket scientist to figure this out. More from Dartmouth, who commissioned the research: 

They also found that test scores represent an especially valuable tool to identify high-achieving applicants from low and middle-income backgrounds; who are first-generation college-bound; as well as students from urban and rural backgrounds.

All the colleges and universities that quickly adopted test-optional admissions in 2020 experienced a surge in applications. Perhaps the push for test-optional was under the guise of woke equality but was nothing more than protecting the bottom line for these institutions. 

A glimpse of sanity returns to woke schools: Admit qualified kids. Next up is corporate America and all tiers of the US government. 

Tyler Durden Mon, 02/05/2024 - 17:20

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Four burning questions about the future of the $16.5B Novo-Catalent deal

To build or to buy? That’s a classic question for pharma boardrooms, and Novo Nordisk is going with both.
Beyond spending billions of dollars to expand…

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To build or to buy? That’s a classic question for pharma boardrooms, and Novo Nordisk is going with both.

Beyond spending billions of dollars to expand its own production capacity for its weight loss drugs, the Danish drugmaker said Monday it will pay $11 billion to acquire three manufacturing plants from Catalent. It’s part of a broader $16.5 billion deal with Novo Holdings, the investment arm of the pharma’s parent group, which agreed to acquire the contract manufacturer and take it private.

It’s a big deal for all parties, with potential ripple effects across the biotech ecosystem. Here’s a look at some of the most pressing questions to watch after Monday’s announcement.

Why did Novo do this?

Novo Holdings isn’t the most obvious buyer for Catalent, particularly after last year’s on-and-off M&A interest from the serial acquirer Danaher. But the deal could benefit both Novo Holdings and Novo Nordisk.

Novo Nordisk’s biggest challenge has been simply making enough of the weight loss drug Wegovy and diabetes therapy Ozempic. On last week’s earnings call, Novo Nordisk CEO Lars Fruergaard Jørgensen said the company isn’t constrained by capital in its efforts to boost manufacturing. Rather, the main challenge is the limited amount of capabilities out there, he said.

“Most pharmaceutical companies in the world would be shopping among the same manufacturers,” he said. “There’s not an unlimited amount of machinery and people to build it.”

While Novo was already one of Catalent’s major customers, the manufacturer has been hamstrung by its own balance sheet. With roughly $5 billion in debt on its books, it’s had to juggle paying down debt with sufficiently investing in its facilities. That’s been particularly challenging in keeping pace with soaring demand for GLP-1 drugs.

Novo, on the other hand, has the balance sheet to funnel as much money as needed into the plants in Italy, Belgium, and Indiana. It’s also struggled to make enough of its popular GLP-1 drugs to meet their soaring demand, with documented shortages of both Ozempic and Wegovy.

The impact won’t be immediate. The parties expect the deal to close near the end of 2024. Novo Nordisk said it expects the three new sites to “gradually increase Novo Nordisk’s filling capacity from 2026 and onwards.”

As for the rest of Catalent — nearly 50 other sites employing thousands of workers — Novo Holdings will take control. The group previously acquired Altasciences in 2021 and Ritedose in 2022, so the Catalent deal builds on a core investing interest in biopharma services, Novo Holdings CEO Kasim Kutay told Endpoints News.

Kasim Kutay

When asked about possible site closures or layoffs, Kutay said the team hasn’t thought about that.

“That’s not our track record. Our track record is to invest in quality businesses and help them grow,” he said. “There’s always stuff to do with any asset you own, but we haven’t bought this company to do some of the stuff you’re talking about.”

What does it mean for Catalent’s customers? 

Until the deal closes, Catalent will operate as a standalone business. After it closes, Novo Nordisk said it will honor its customer obligations at the three sites, a spokesperson said. But they didn’t answer a question about what happens when those contracts expire.

The wrinkle is the long-term future of the three plants that Novo Nordisk is paying for. Those sites don’t exclusively pump out Wegovy, but that could be the logical long-term aim for the Danish drugmaker.

The ideal scenario is that pricing and timelines remain the same for customers, said Nicole Paulk, CEO of the gene therapy startup Siren Biotechnology.

Nicole Paulk

“The name of the group that you’re going to send your check to is now going to be Novo Holdings instead of Catalent, but otherwise everything remains the same,” Paulk told Endpoints. “That’s the best-case scenario.”

In a worst case, Paulk said she feared the new owners could wind up closing sites or laying off Catalent groups. That could create some uncertainty for customers looking for a long-term manufacturing partner.

Are shareholders and regulators happy? 

The pandemic was a wild ride for Catalent’s stock, with shares surging from about $40 to $140 and then crashing back to earth. The $63.50 share price for the takeover is a happy ending depending on the investor.

On that point, the investing giant Elliott Investment Management is satisfied. Marc Steinberg, a partner at Elliott, called the agreement “an outstanding outcome” that “clearly maximizes value for Catalent stockholders” in a statement.

Elliott helped kick off a strategic review last August that culminated in the sale agreement. Compared to Catalent’s stock price before that review started, the deal pays a nearly 40% premium.

Alessandro Maselli

But this is hardly a victory lap for CEO Alessandro Maselli, who took over in July 2022 when Catalent’s stock price was north of $100. Novo’s takeover is a tacit acknowledgment that Maselli could never fully right the ship, as operational problems plagued the company throughout 2023 while it was limited by its debt.

Additional regulatory filings in the next few weeks could give insight into just how competitive the sale process was. William Blair analysts said they don’t expect a competing bidder “given the organic investments already being pursued at other leading CDMOs and the breadth and scale of Catalent’s operations.”

The Blair analysts also noted the companies likely “expect to spend some time educating relevant government agencies” about the deal, given the lengthy closing timeline. Given Novo Nordisk’s ascent — it’s now one of Europe’s most valuable companies — paired with the limited number of large contract manufacturers, antitrust regulators could be interested in taking a close look.

Are Catalent’s problems finally a thing of the past?

Catalent ran into a mix of financial and operational problems over the past year that played no small part in attracting the interest of an activist like Elliott.

Now with a deal in place, how quickly can Novo rectify those problems? Some of the challenges were driven by the demands of being a publicly traded company, like failing to meet investors’ revenue expectations or even filing earnings reports on time.

But Catalent also struggled with its business at times, with a range of manufacturing delays, inspection reports and occasionally writing down acquisitions that didn’t pan out. Novo’s deep pockets will go a long way to a turnaround, but only the future will tell if all these issues are fixed.

Kutay said his team is excited by the opportunity and was satisfied with the due diligence it did on the company.

“We believe we’re buying a strong company with a good management team and good prospects,” Kutay said. “If that wasn’t the case, I don’t think we’d be here.”

Amber Tong and Reynald Castañeda contributed reporting.

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Petrina Kamya, Ph.D., Head of AI Platforms at Insilico Medicine, presents at BIO CEO & Investor Conference

Petrina Kamya, PhD, Head of AI Platforms and President of Insilico Medicine Canada, will present at the BIO CEO & Investor Conference happening Feb….

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Petrina Kamya, PhD, Head of AI Platforms and President of Insilico Medicine Canada, will present at the BIO CEO & Investor Conference happening Feb. 26-27 at the New York Marriott Marquis in New York City. Dr. Kamya will speak as part of the panel “AI within Biopharma: Separating Value from Hype,” on Feb. 27, 1pm ET along with Michael Nally, CEO of Generate: Biomedicines and Liz Schwarzbach, PhD, CBO of BigHat Biosciences.

Credit: Insilico Medicine

Petrina Kamya, PhD, Head of AI Platforms and President of Insilico Medicine Canada, will present at the BIO CEO & Investor Conference happening Feb. 26-27 at the New York Marriott Marquis in New York City. Dr. Kamya will speak as part of the panel “AI within Biopharma: Separating Value from Hype,” on Feb. 27, 1pm ET along with Michael Nally, CEO of Generate: Biomedicines and Liz Schwarzbach, PhD, CBO of BigHat Biosciences.

The session will look at how the latest artificial intelligence (AI) tools – including generative AI and large language models – are currently being used to advance the discovery and design of new drugs, and which technologies are still in development. 

The BIO CEO & Investor Conference brings together over 1,000 attendees and more than 700 companies across industry and institutional investment to discuss the future investment landscape of biotechnology. Sessions focus on topics such as therapeutic advancements, market outlook, and policy priorities.

Insilico Medicine is a leading, clinical stage AI-driven drug discovery company that has raised over $400m in investments since it was founded in 2014. Dr. Kamya leads the development of the Company’s end-to-end generative AI platform, Pharma.AI from Insilico’s AI R&D Center in Montreal. Using modern machine learning techniques in the context of chemistry and biology, the platform has driven the discovery and design of 30+ new therapies, with five in clinical stages – for cancer, fibrosis, inflammatory bowel disease (IBD), and COVID-19. The Company’s lead drug, for the chronic, rare lung condition idiopathic pulmonary fibrosis, is the first AI-designed drug for an AI-discovered target to reach Phase II clinical trials with patients. Nine of the top 20 pharmaceutical companies have used Insilico’s AI platform to advance their programs, and the Company has a number of major strategic licensing deals around its AI-designed therapeutic assets, including with Sanofi, Exelixis and Menarini. 

 

About Insilico Medicine

Insilico Medicine, a global clinical stage biotechnology company powered by generative AI, is connecting biology, chemistry, and clinical trials analysis using next-generation AI systems. The company has developed AI platforms that utilize deep generative models, reinforcement learning, transformers, and other modern machine learning techniques for novel target discovery and the generation of novel molecular structures with desired properties. Insilico Medicine is developing breakthrough solutions to discover and develop innovative drugs for cancer, fibrosis, immunity, central nervous system diseases, infectious diseases, autoimmune diseases, and aging-related diseases. www.insilico.com 


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