Thalamus

NRx Pharmaceuticals Announces the Licensure of a US Patent to Support Use of NRX-101™ for Chronic Pain

Retrieved on: 
Monday, August 7, 2023

The patent is supported by extensive nonclinical data and early clinical data that suggest the potential for NMDA antagonist drugs, such as NRX-101 to decrease both chronic pain and neuropathic pain while potentially decreasing craving for opioids.

Key Points: 
  • The patent is supported by extensive nonclinical data and early clinical data that suggest the potential for NMDA antagonist drugs, such as NRX-101 to decrease both chronic pain and neuropathic pain while potentially decreasing craving for opioids.
  • "We delighted welcome Dr. Apkarian to the NRx SAB," said Jonathan Javitt, Chief Scientist and Founder of NRx Pharmaceuticals.
  • "Adding a scientist of Vania's caliber will be invaluable to advancing our clinical program in Chronic Pain."
  • In June 2023, the Company announced a focus on chronic pain as the next focus on NRX-101's development.

UT Health Science Center San Antonio develops tool that counts brain lesions in seconds

Retrieved on: 
Friday, July 14, 2023

SAN ANTONIO, July 13, 2023 /PRNewswire-PRWeb/ -- An artificial intelligence (AI) tool developed at The University of Texas Health Science Center at San Antonio accurately counts brain lesions on MRI scans within seconds. Once it is adapted to the clinic, the AI tool should help neuroradiologists to evaluate patients' brain diseases at earlier stages.

Key Points: 
  • An innovative deep-learning tool developed by researchers at multiple institutions including The University of Texas Health Science Center at San Antonio promises to change this.
  • SAN ANTONIO, July 13, 2023 /PRNewswire-PRWeb/ -- An artificial intelligence (AI) tool developed at The University of Texas Health Science Center at San Antonio accurately counts brain lesions on MRI scans within seconds.
  • The AI tool leverages the power of UT Health Science Center San Antonio's Genie supercomputer, Habes said.
  • The University of Texas Health Science Center at San Antonio (UT Health San Antonio), a primary driver for San Antonio's $44.1 billion health care and biosciences sector, is the largest academic research institution in South Texas with an annual research portfolio of more than $360 million.

Orchard Therapeutics Announces Positive Clinical and Preclinical Data in Programs Targeting Neurometabolic and CNS Disorders at ASGCT

Retrieved on: 
Friday, May 19, 2023

BOSTON and LONDON, May 19, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced multiple clinical and preclinical updates from its portfolio of investigational hematopoietic stem cell (HSC) gene therapies in neurometabolic and neurodegenerative disorders. The data are being featured in several oral presentations at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles.

Key Points: 
  • The data are being featured in several oral presentations at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles.
  • At baseline, most patients presented with severe joint range of motion impairment, severe acetabular (hip) dysplasia and varying degrees of dorso-lumbar kyphosis.
  • Treatment with OTL-203 was generally well tolerated and demonstrated extensive metabolic correction over four years after treatment.
  • Data from in vivo studies indicate effective GRN protein delivery to the CNS of knockout mice transplanted with gene-modified HSCs.

Capsida Biotherapeutics to Present New Data Showing Increased Capsid Expression in CNS at American Society of Gene & Cell Therapy 26th Annual Meeting

Retrieved on: 
Wednesday, May 10, 2023

THOUSAND OAKS, Calif., May 10, 2023 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") today announced it will present preliminary data on the company's next-generation engineered adeno-associated virus (AAV) capsids in non-human primates. The initial data showed that Capsida's engineered AAV capsids, when delivered intravenously at a clinically relevant dose, resulted in widespread transduction including protein production across multiple brain regions including cortex, thalamus, and putamen, in up to 68% of neurons. Importantly, liver transduction was reduced five-fold compared to wild-type AAVs.

Key Points: 
  • Capsida will present these data at an industry-sponsored symposium on Friday, May 19, 2023 at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, being held May 16-20, 2023 in Los Angeles.
  • In addition, Capsida will present several posters that highlight optimization of process and analytical methods for engineered AAV capsids as part of the ASGCT meeting.
  • "This capsid performance represents the potential to achieve a significantly increased therapeutic index," said Susan Catalano, Chief Scientific Officer of Capsida.
  • Capsida will present three posters that highlight the optimization of process and analytical development methods for engineered AAV capsids.

Concussion: almost half of people still show signs of brain injury after six months

Retrieved on: 
Tuesday, May 2, 2023

Previous research found that clinicians estimated one in ten people might experience long-term symptoms after a concussion.

Key Points: 
  • Previous research found that clinicians estimated one in ten people might experience long-term symptoms after a concussion.
  • Our study, published in Brain, found that almost half of people who suffered a concussion had not fully recovered six months after their injury.
  • To conduct our study, we analysed brain scans from over 100 patients who had recently experienced concussion from all across Europe.
  • This allows us to understand if the brain is functioning as it should or if there are problems with a person’s brain connectivity.
  • We also found that almost half of people with concussion had not fully recovered six months after the injury.

Concussion treatment

    • Our research makes an important step in understanding concussion, showing that even a single brain injury can have clear consequences in some people.
    • This not only shows us how concussion affects people differently, it may also give us targets we can use to develop drugs that alleviate concussion symptoms.
    • Although there’s still a lot we don’t know about concussion – including the effects that repetitive concussion may have on the brain – it’s promising to see this condition is being taken more seriously, especially in sports where it can be common.
    • New UK guidelines for grassroots sports like football and rugby now require players to sit out of play for at least 24 hours after a suspected concussion, which may help to prevent concussion and improve recovery after one.

ClearPoint Neuro Extends Software License and Research Agreement with Philips to Include Fully Automatic Subnuclei Segmentation for DBS

Retrieved on: 
Tuesday, April 11, 2023

The design goal of the ClearPoint Maestro® Brain Model is to enable highly accurate ClearPoint interventions under both MRI and CT guidance by facilitating patient-specific, multi-modal fusion for accurate target identification and trajectory planning.

Key Points: 
  • The design goal of the ClearPoint Maestro® Brain Model is to enable highly accurate ClearPoint interventions under both MRI and CT guidance by facilitating patient-specific, multi-modal fusion for accurate target identification and trajectory planning.
  • “Extending our software collaboration with Philips beyond the subcortical brain regions in Maestro to include segmentation of subnuclei from MRI is key to our planned expansion into the operating room,” commented Joe Burnett, President and CEO of ClearPoint Neuro.
  • The unique methodology of the brain model combines deformable surfaces with active shape models and machine learning.
  • Maestro was FDA cleared in 2022 and is currently in limited market release at select U.S. academic medical centers and in pharmaceutical clinical trials.

Avidity Biosciences Provides Regulatory Update on AOC 1001 for Myotonic Dystrophy Type 1 and Plans to Present Top-line Data from Phase 1/2 MARINA™ Trial at AAN Annual Meeting

Retrieved on: 
Thursday, March 30, 2023

SAN DIEGO, March 30, 2023 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today provided an update on the Phase 1/2 MARINA™ trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1), an underrecognized, progressive and often fatal neuromuscular disease with no approved treatment options. Discussions are ongoing with the U.S. Food and Drug Administration (FDA) regarding the partial clinical hold on new participant enrollment as Avidity continues to provide new AOC 1001 data as it emerges from the MARINA trial. AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA.

Key Points: 
  • AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA.
  • "We are continuing to work diligently with the FDA and remain very confident in AOC 1001 and its benefit/risk profile.
  • We look forward to reporting safety and functional top-line data from the MARINA trial at AAN next month."
  • Data from the preliminary assessment of AOC 1001 supports that the targeted dose range is between 2 mg/kg and 4 mg/kg.

Tiziana Life Sciences Ltd (“Tiziana” or “the Company”) - Interim Results for the Six Months Ended 30 June 2022

Retrieved on: 
Tuesday, December 27, 2022

LONDON, Dec. 27, 2022 (GLOBE NEWSWIRE) -- Tiziana Life Sciences Ltd (“Tiziana”, NASDAQ: TLSA), a biotechnology company focused on innovative therapeutics for oncology, inflammation, and infectious diseases today announces its interim results for the six months ended 30 June 2022.

Key Points: 
  • LONDON, Dec. 27, 2022 (GLOBE NEWSWIRE) -- Tiziana Life Sciences Ltd (“Tiziana”, NASDAQ: TLSA), a biotechnology company focused on innovative therapeutics for oncology, inflammation, and infectious diseases today announces its interim results for the six months ended 30 June 2022.
  • These protocol amendments or revisions are intended to expedite patient enrollment with study completion targeted for the fourth quarter of 2022.
  • These results confirm the previously reported data, from the first SPMS patient after three months of treatment of the first SPMS patient.
  • For the six months to 30 June 2022 the consolidated Group made a loss of $8.3m (six months to 30 June 2021: $17.0m).

Oncotelic Initiates Clinical Trials Evaluating OT-101 against Pediatric Gliomas

Retrieved on: 
Tuesday, November 8, 2022

OT-101 is a first-in-class anti-TGF-2 ribonucleic acid ("RNA") therapeutic that has exhibited single agent activity in relapsed/refractory cancer patients in multiple clinical trials.

Key Points: 
  • OT-101 is a first-in-class anti-TGF-2 ribonucleic acid ("RNA") therapeutic that has exhibited single agent activity in relapsed/refractory cancer patients in multiple clinical trials.
  • Therefore, there is an urgent need for therapeutic innovations for treatment of pediatric DMG, explained Fatih Uckun, MD PhD, the Chief Medical Officer of Oncotelic.
  • "This is the first of a series of planned clinical trials in pediatric patients with gliomas evaluating clinical benefit while also assessing predictive biomarkers."
  • "The groundwork laid down by our successful clinical program in adult gliomas and deep datamining by the team will now help guide the development of OT-101 for pediatric DMG.

Thalamus.Org Launches to Address Inequities in the Physician Workforce

Retrieved on: 
Tuesday, September 27, 2022

NASHVILLE, Tenn., Sep. 27, 2022 /PRNewswire-PRWeb/ -- Thalamus.Org (https://thalamus.org), a 501(c)(3) nonprofit organization, announced its official launch today. Its mission is to make the medical residency selection, placement, and transition process affordable, fair, and accessible to all medical students.

Key Points: 
  • Its mission is to make the medical residency selection, placement, and transition process affordable, fair, and accessible to all medical students.
  • The goal is to close the disparate financial gap and minimize the additional hurdles such students have in navigating the residency application process.
  • "We've intentionally taken a broad approach of defining underrepresented in medicine," said Jason Reminick, MD, MBA, MS, Founder and Chair of Thalamus.Org.
  • "As a health funder in the most racially diverse state in the US, we are thrilled to help build a physician workforce that looks more like California's population."