NOS1

Solid Biosciences Receives Rare Pediatric Disease Designation from the FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003

Retrieved on: 
Monday, April 1, 2024
Patient, DNA, Hope, Tropism, Duchenne muscular dystrophy, Duchenne, NOS1, Safety, FDA, DMD, Food, Pharmaceutical industry

CHARLESTOWN, Mass., April 01, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.

Key Points: 
  • “Solid’s receipt of Rare Pediatric Disease Designation for SGT-003 highlights the continuing need for transformational treatments for this devastating disease,” said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences.
  • “The key components of SGT-003 were rationally designed to improve on first generation gene therapies to provide skeletal muscle tropism, enhanced durability, and improved clinical outcomes.
  • nNOS is believed to play a crucial role in both muscular function and endurance,” said Dr. Gabriel Brooks, M.D., Chief Medical Officer at Solid Biosciences.
  • “We look forward to rapidly bringing SGT-003 to the clinic and hope to all Duchenne patients in need.”

Beyond Air® Reports Financial Results for Third Quarter of Fiscal Year 2024

Retrieved on: 
Monday, February 12, 2024
Nitrogen oxide, Conference call, Nitric oxide, Research, PMA, B cell, Hebrew University of Jerusalem, FDA, Clinical research associate, SITC, NOS1, UNO, Pneumonia, Season, ASD, Society, Conference, Commercial, HUJI, Phenotype, Patient, DSM-IV codes, Immunotherapy, VCAP, NTM, Fiscal year, Nontuberculous mycobacteria

“In the third quarter of fiscal year 2024, we reported solid sequential revenue growth of more than 60% compared with the second quarter.

Key Points: 
  • “In the third quarter of fiscal year 2024, we reported solid sequential revenue growth of more than 60% compared with the second quarter.
  • This increasing demand for our current LungFit PH system was seen even as we prepared to launch a key software update.
  • As our inventory of upgraded LungFit PH grows, we expect to be able to meet accelerating demand over the next several quarters.
  • General and administrative expenses for the three months ended December 31, 2023 and December 31, 2022 were $9.8 million and $8.9 million, respectively.

Solid Biosciences Granted FDA Orphan Drug Designation for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003

Retrieved on: 
Tuesday, January 16, 2024
Patient, Duchenne, Solid, Nitric oxide synthase, ODD, IRB, Food, Capsid, FDA, NOS1, Duchenne muscular dystrophy, Clinical trial, Pharmaceutical industry

CHARLESTOWN, Mass., Jan. 16, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it has been granted orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.

Key Points: 
  • CHARLESTOWN, Mass., Jan. 16, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it has been granted orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.
  • “Obtaining ODD status for SGT-003, along with Fast Track Designation granted last month, furthers our efforts to meet the ongoing challenge of treating this devastating disease as expeditiously as possible,” said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences.
  • “SGT-003 therapy stands out among other Duchenne gene therapy candidates by leveraging a novel capsid and a muscle tropic vector delivering a microdystrophin that incorporates a neuronal Nitric Oxide Synthase (nNOS) binding domain.
  • “Obtaining ODD status is an exciting development that we believe will aid our efforts to bring advanced treatment options to those patients affected by Duchenne.”

Locanabio Announces Presentation of Preclinical Data Demonstrating an Improved Vectorized snRNA Platform with Applications in Exon Skipping for the Potential Treatment of Duchenne Muscular Dystrophy (DMD) at the 28th Annual Congress of the World Muscle So

Retrieved on: 
Friday, October 6, 2023
Genome, RNA, DMD, Therapy, NOS1, IND, Exon, AAV, SnRNA-seq, SAN, ADAR, Tissue, Messenger, Sarcolemma, Short interspersed nuclear element, Asos, Patient, Poster, Reading, Vaccine, Publication

“As these data demonstrate, our vectorized snRNA platform combines the therapeutic potential of exon skipping with the delivery advantages of an AAV gene therapy,” said John Leonard, Ph.D., Locanabio’s chief scientific officer.

Key Points: 
  • “As these data demonstrate, our vectorized snRNA platform combines the therapeutic potential of exon skipping with the delivery advantages of an AAV gene therapy,” said John Leonard, Ph.D., Locanabio’s chief scientific officer.
  • In contrast, exon skipping enables the production of a near full-length dystrophin from the native transcript expressed in affected tissues.
  • However, prior exon skipping approaches with first generation antisense oligonucleotides (ASOs) have been limited by poor biodistribution and tolerability resulting in negligible restoration of dystrophin protein.
  • “The ability to package multiple snRNA cassettes amplifies potency relative to delivery of single ASOs for exon skipping.

Beyond Air® Announces Publication of Positive New Preclinical Data for the Treatment of Autism Spectrum Disorder

Retrieved on: 
Monday, September 18, 2023
Faculty, Injection, Al-Quds, Hebrew University of Jerusalem, Medical school, Therapy, ScienceDirect, Doctor of Philosophy, Social, Pharming, Autism spectrum, HUJI, Nitric oxide, DSM-IV codes, Research, NOS1, Behavior, Assistant, UCL, Plasma, NO, Patient, ASD, School, Spectrum, Effect, Memory, Nitrogen oxide, Phenotype, Yissum Research Development Company of the Hebrew University, Pharmaceutical industry, Pharmacy, Medicine

GARDEN CITY, N.Y., Sept. 18, 2023 (GLOBE NEWSWIRE) -- Beyond Air, Inc. (NASDAQ: XAIR) (“Beyond Air” or the “Company”) a commercial stage medical device and biopharmaceutical company focused on harnessing the power of endogenous and exogenous nitric oxide (NO) to improve the lives of patients suffering from respiratory illnesses, neurological disorders and solid tumors (through its affiliate Beyond Cancer, Ltd. (“Beyond Cancer”)), today announced the publication of new data that support ongoing preclinical development of selective nNOS inhibitors for the treatment of Autism Spectrum Disorder (ASD).

Key Points: 
  • The slow-release formulation led to reversal of behavioral phenotypes and the nitrosative stress of the Shank3 mouse model of autism.
  • The Hebrew University of Jerusalem (HUJI) has previously published studies suggesting that NO is a pathologic factor in ASD.
  • In previously published studies, 10-day daily injections of 7-nitroindazole (7-NI), a selective nNOS inhibitor, reversed the manifestations of ASD phenotype, and reversed nitrosative stress.
  • “These novel data provide new evidence of the role of nitric oxide in autism.

China Medical System Gained Exclusive License of an Anti-Ischemic Stroke Class 1 Innovative Drug

Retrieved on: 
Thursday, August 24, 2023
Agreement, Post-stroke depression, CMS, Central nervous system, Marketing, Stroke, Y-3, NOS1, DSM-IV codes, Tablet, Incidence, Patient, Health, Phase, Family, Safety, Pharmaceutical industry

Y-3, a Class 1 Innovative Drug injection, is used to alleviate neurological symptoms and dysfunction of daily activities caused by acute ischemic stroke.

Key Points: 
  • Y-3, a Class 1 Innovative Drug injection, is used to alleviate neurological symptoms and dysfunction of daily activities caused by acute ischemic stroke.
  • After approved for marketing in China, it is expected to meet the clinical needs of drug with both safety and efficacy, providing new treatment options to ischemic stroke patients in China.
  • Y-3 Injection is a Class 1 innovative drug - small molecule compound, which is used to alleviate neurological symptoms and dysfunction of daily activities caused by acute ischemic stroke.
  • For further detailed information, please visit the following link for the announcement , “Voluntary and Business Update Announcement Gaining Exclusive License of an Anti-Ischemic Stroke Class 1 Innovative Drug” issued by CMS on August 24 2023.

Beyond Air® Reports Financial Results for First Quarter of Fiscal Year 2024

Retrieved on: 
Thursday, August 10, 2023
COPD, Advanced Science, UNO, Conference call, Spectrum, Calendar, DSM-IV codes, NOS1, Avenue Capital Group, Trial of the century, Hospital, NTM, Nontuberculous mycobacteria, Patient, Atmosphere of Earth, ASD, VCAP, Research, FDA, Conference, Pneumonia, Vaccine, Pharmaceutical industry, Cryptocurrency

“We concluded the first quarter of our fiscal year 2024 with LungFit PH adopted as the nitric oxide system of choice at several hospitals in the United States.

Key Points: 
  • “We concluded the first quarter of our fiscal year 2024 with LungFit PH adopted as the nitric oxide system of choice at several hospitals in the United States.
  • Our commercial team has implemented training programs and support at each of these hospitals, and report that LungFit PH has been well received.
  • General and administrative expenses for the three months ended June 30, 2023 and June 30, 2022 were $10.9 million and $8.2 million, respectively.
  • As of June 30, 2023, the Company had cash, cash equivalents and marketable securities of $57.0 million and $2.7 million in restricted cash.

Beyond Air® Announces Positive Preclinical Data on Role of Nitric Oxide in Autism Spectrum Disorder (ASD)

Retrieved on: 
Monday, August 7, 2023
Faculty, Al-Quds, Hebrew University of Jerusalem, Medical school, Autism spectrum, GABAergic, HUJI, Nitric oxide, DSM-IV codes, ESN, Mutation, Brain, Research, NOS1, Assistant, ISN, UCL, CNTNAP2, NO, Poster, Patient, ASD, European Society for Neurochemistry, School, Spectrum, Mouse, European Society for Primary Immunodeficiencies, Yissum Research Development Company of the Hebrew University, Pharmaceutical industry, Pharmacy, Medicine

GARDEN CITY, N.J., Aug. 07, 2023 (GLOBE NEWSWIRE) -- Beyond Air, Inc. (NASDAQ: XAIR) (“Beyond Air” or the “Company”) a commercial stage medical device and biopharmaceutical company focused on harnessing the power of endogenous and exogenous nitric oxide (NO) to improve the lives of patients suffering from respiratory illnesses, neurological disorders and solid tumors (through its affiliate Beyond Cancer, Ltd. (“Beyond Cancer”)), today announced the presentation of data that support the ongoing preclinical development of selective neuronal nitric oxide synthase (nNOS) inhibibitors for the treatment of Autism Spectrum Disorder (ASD). These data will be presented by the Amal Lab from the Hebrew University of Jerusalem (HUJI) in a poster at the Annual International Society of Neurochemistry (ISN) and the European Society of Neurochemistry (ESN) meetings, which are being held in Portugal from August 8th - 11th.

Key Points: 
  • Research conducted recently at HUJI has shown that mutation in the CNTNAP2 gene increases NO formation in the brain of the ASD mouse model.
  • These data demonstrate that the inhibition of NO production may reverse ASD phenotypes, and provide further evidence that NO is a pathologic factor in autism.
  • These results have led researchers to believe that NO is a novel target for the treatment of ASD and other neurological disorders.
  • The preclinical data developed thus far show that this exciting novel approach to treating ASD has the potential to address a significant unmet medical need.

Beyond Air® Reports Financial Results for Fourth Quarter and Fiscal Year-End 2023

Retrieved on: 
Thursday, June 22, 2023
COPD, Bronchiolitis, Advanced Science, Autism spectrum, Conference call, Calendar, Nitric oxide, DSM-IV codes, GAAP, Research, NOS1, Avenue Capital Group, PPHN, NO, Hospital, NTM, Nontuberculous mycobacteria, Patent, Patient, ASD, VCAP, Spectrum, Delivery, BRO, FDA, Conference, Pharmaceutical industry, Cryptocurrency, Cancer, Circassia

Selling, general and administrative expenses for the fiscal year ended March 31, 2023, increased to $34.7 million, compared with $18.4 million for the fiscal year ended March 31, 2022.

Key Points: 
  • Selling, general and administrative expenses for the fiscal year ended March 31, 2023, increased to $34.7 million, compared with $18.4 million for the fiscal year ended March 31, 2022.
  • As a reminder, the Company paid $2.5 million to Circassia in the 2nd fiscal quarter of 2023, we have another $3.5 million to pay in the 2nd fiscal quarter of 2024 with the final $4.5 million not due until the 2nd quarter of fiscal 2025.
  • Other income and expense was a $7.3 million loss for the fiscal year ended March 31, 2023, compared with a $3.4 million loss for the fiscal year ended March 31, 2022.
  • The increase of $3.9 million was primarily due to a non-product related legal matter that was resolved in the fourth fiscal quarter of 2023.

Beyond Air® Licenses Commercial Rights to Selective Neuronal Nitric Oxide Synthase (nNOS) Inhibitors for the Treatment of Autism Spectrum Disorder from Hebrew University of Jerusalem

Retrieved on: 
Thursday, June 15, 2023
Al-Quds, Medical school, HUJI, Nitric oxide, Wolf Foundation, NOS1, Doctor of Philosophy, DSM-IV codes, NO, Journal, Patient, Society, Phenotype, Male, Human, Hebrew University of Jerusalem, Advanced Science, Research, SNAP, FDA, Conference, Neuropharmacology, Conference call, Cancer, MIT, Biomarker, Cell, Publication, ASD, Webcast, LTD, Faculty, Partnership, Cell death, Spectrum, Urinary tract infection, SHANK3, Atmosphere of Earth, University of Toronto Faculty of Medicine, Yissum Research Development Company of the Hebrew University, Mouse, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, M1, Psychiatry, Pharmacy, Tel Aviv University, Medicine

GARDEN CITY, N.Y., June 15, 2023 (GLOBE NEWSWIRE) -- Beyond Air, Inc. (NASDAQ: XAIR) (“Beyond Air” or the “Company”), a commercial stage medical device and biopharmaceutical company focused on harnessing the power of endogenous and exogenous nitric oxide (NO) to improve the lives of patients suffering from respiratory illnesses, neurological disorders and solid tumors (through its affiliate Beyond Cancer, Ltd.), today announced that it has entered into an agreement with Yissum Research Development Company of the Hebrew University of Jerusalem, LTD. to license the commercial rights for multiple neuronal nitric oxide synthase (nNOS) inhibitor candidates, currently being developed for the treatment of autism spectrum disorder (ASD) and other neurological conditions. There are currently no therapies approved by the U.S. Food and Drug Administration (FDA) specifically for the treatment of ASD.

Key Points: 
  • There are currently no therapies approved by the U.S. Food and Drug Administration (FDA) specifically for the treatment of ASD.
  • The partial inhibition of nNOS reduces nitrosative stress biomarkers and reverses the molecular, synaptic and behavioral ASD-associated phenotypes.
  • Dr. Amal is an Assistant Professor at the Institute for Drug Research, Faculty of Medicine at the Hebrew University of Jerusalem.
  • He began his academic journey at the age of seventeen, with a degree in Pharmacy, from the Hebrew University.