BAG3

Affinia Therapeutics to Present Data on Novel Cardiotropic and BBB-Penetrant AAV Capsids and Preclinical Efficacy and Safety in Genetic Cardiomyopathies and Sporadic ALS at the American Society of Gene & Cell Therapy 2024 Annual Meeting

Retrieved on: 
Monday, April 22, 2024
DRG, Heart, Traffic barrier, Computational science, Safety, Abstract (summary), Tropism, Cardiac muscle, MD, Structure, Diagram, Cell, BAG3, AAV, Skeletal muscle, CNS, Senior, Synthetic media, Liver, Gene, Society, ALS, Ataxin-2, BBB, Toxicity, Central nervous system, Messenger, Generative, Transcytosis, Annual general meeting, Therapy, Vaccine

Affinia's novel capsids have favorable manufacturing yields and levels of preexisting population immunity.

Key Points: 
  • Affinia's novel capsids have favorable manufacturing yields and levels of preexisting population immunity.
  • "We are excited to unveil data on our next-generation bespoke capsids that demonstrate robust cardiac transduction while detargeting the liver and DRG.
  • One oral presentation showcases data from Affinia's novel, BBB-penetrant AAV capsid with a potential therapeutic application to sporadic ALS.
  • The first poster showcases data that confirm the novel cardiotropic AAV capsid's safety and therapeutic efficacy in an animal model of cardiac dysfunction with a potential therapeutic application in genetic cardiomyopathies.

New Chinese medicine drug developed by HKBU for myofibrillar myopathy granted orphan drug designation by FDA

Retrieved on: 
Wednesday, January 10, 2024
Fruit, Patient, Rosaceae, Research, Facial muscles, Associate, Delayed onset muscle soreness, BAG3, Carica, Hong Kong Baptist University, Muscular dystrophy, Myopathy, Disability studies, Prescription, HKBU, Traditional Chinese medicine, Disease, Safety, FDA, Pain, Caricaceae, Investigational New Drug, Agonal respiration, Cardiomyopathy, Paralysis, Muscle atrophy, Pharmaceutical industry

The drug has obtained the orphan drug designation from the US Food and Drug Administration (FDA), and is the first botanical drug in Hong Kong to receive this qualification.

Key Points: 
  • The drug has obtained the orphan drug designation from the US Food and Drug Administration (FDA), and is the first botanical drug in Hong Kong to receive this qualification.
  • The research team plans to submit an Investigational New Drug application to FDA in two years to conduct clinical trials.
  • Professor Bian Zhaoxiang, Associate Vice-President (Chinese Medicine Development) and Director of the Centre for Chinese Herbal Medicine Drug Development at HKBU, said: "CDD-2107 is the fruit of the integration of Chinese medicine theory and modern technology.
  • Receiving the orphan drug designation by FDA for CDD-2107 is an encouraging milestone and recognition of our team's dedication to scientific innovation."

New Chinese medicine drug developed by HKBU for myofibrillar myopathy granted orphan drug designation by FDA

Retrieved on: 
Wednesday, January 10, 2024
Fruit, Patient, Rosaceae, Research, Facial muscles, Associate, Delayed onset muscle soreness, BAG3, Carica, Multimedia, Hong Kong Baptist University, Muscular dystrophy, Myopathy, Disability studies, Prescription, HKBU, Traditional Chinese medicine, Disease, Safety, FDA, Pain, Caricaceae, Investigational New Drug, Agonal respiration, Cardiomyopathy, Paralysis, Muscle atrophy, Pharmaceutical industry

The drug has obtained the orphan drug designation from the US Food and Drug Administration (FDA), and is the first botanical drug in Hong Kong to receive this qualification.

Key Points: 
  • The drug has obtained the orphan drug designation from the US Food and Drug Administration (FDA), and is the first botanical drug in Hong Kong to receive this qualification.
  • The research team plans to submit an Investigational New Drug application to FDA in two years to conduct clinical trials.
  • Professor Bian Zhaoxiang, Associate Vice-President (Chinese Medicine Development) and Director of the Centre for Chinese Herbal Medicine Drug Development at HKBU, said: "CDD-2107 is the fruit of the integration of Chinese medicine theory and modern technology.
  • Receiving the orphan drug designation by FDA for CDD-2107 is an encouraging milestone and recognition of our team's dedication to scientific innovation."

Solid Biosciences Provides Second Quarter Business Update and Financial Results

Retrieved on: 
Monday, August 14, 2023
Insurance, Duchenne muscular dystrophy, Ataxia, Solid, Research, Duchenne, Sale, BAG3, Investigational New Drug, Patient, Investment, IND, Pharmaceutical industry, Video game, Birth control

CHARLESTOWN, Mass., Aug. 14, 2023 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the second quarter ended June 30, 2023, and provided a business update.

Key Points: 
  • “We made strong progress this quarter across multiple programs in our pipeline of genetic medicines and remain on track to submit an IND for SGT-003 in the fourth quarter of this year,” said Bo Cumbo, President and CEO of Solid Biosciences.
  • Solid also continues to advance preclinical programs in both cardiac and neuromuscular indications as well as platform enabling capsids.
  • There were no collaboration revenues for the second quarter of 2023, compared to $6.2 million, for the second quarter of 2022.
  • Net loss for the second quarter of 2023 was $24.6 million, compared to $25.1 million for the second quarter of 2022.

Rocket Pharmaceuticals Reports Second Quarter 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Thursday, August 10, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, University of California, San Diego Performance-Based Skills Assessment, Communication, Advanced Therapy Medicinal Product, LV, University, PKP2, Marketing, European Medicines Agency, Survival, Clinical trial, Bone marrow, UCSD, Arrhythmia, AAV, Disorder, Medication, Fast track (FDA), EMA, ATMP, MMC, BAG3, Investigational New Drug, Invitae, Patient, IRB, Investment, CGMP, LAMP2, Genetic testing, Cardiomyopathy, FDA, Death, BLA, IND, Gene, Safety, Pharmaceutical industry, Vaccine, Fine chemical, Cryptocurrency, G&A, Danon disease

“The second quarter of 2023 marked a period of strong forward momentum across our cardiovascular AAV and hematology LV portfolios highlighted by the BLA submission for our LAD-I program, Rocket’s first product filing.

Key Points: 
  • “The second quarter of 2023 marked a period of strong forward momentum across our cardiovascular AAV and hematology LV portfolios highlighted by the BLA submission for our LAD-I program, Rocket’s first product filing.
  • ATMP designation is intended for medicines that offer groundbreaking new opportunities and allows for a single evaluation and authorization procedure.
  • Initiating two-patient pediatric safety run-in for RP-A501 pivotal study; approaching final alignment with FDA on primary composite endpoint to support accelerated approval.
  • As of June 30, 2023, Rocket had cash, cash equivalents and investments of $307.0 million.

Solid Biosciences Provides First Quarter Business Update and Financial Results

Retrieved on: 
Thursday, May 11, 2023
Duchenne muscular dystrophy, Gene, Ataxia, Solid, Research, Duchenne, Sale, BAG3, Investigational New Drug, DMD, Patient, Investment, Cell, IND, Society, Pharmaceutical industry, Cryptocurrency

CHARLESTOWN, Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the first quarter ended March 31, 2023, and provided a business update.

Key Points: 
  • “In the first quarter of 2023, we made strong progress across multiple programs in our diversified pipeline of genetic medicines and remain on track to achieve important milestones this year,” said Bo Cumbo, President, and CEO of Solid Biosciences.
  • There were no collaboration revenues for the first quarter of 2023, compared to $1.9 million, for the first quarter of 2022.
  • Net loss for the first quarter of 2023 was $30.1 million, compared to $25.3 million for the first quarter of 2022.
  • The Company expects that its cash, cash equivalents, and available-for-sale securities will enable it to fund key strategic priorities into 2025.

Solid Biosciences Outlines Strategy for Leadership in Precision Genetic Medicines with Focus on Neuromuscular and Cardiac Diseases

Retrieved on: 
Tuesday, January 10, 2023
IND, Disease, Spinal cord, CFA, Tissue, Heart, AAV, Duchenne muscular dystrophy, FA, HSV, Solid, Conference, Capsid, Cerebellum, Tropism, NHP, Investigational New Drug, Duchenne, Research, Heart failure, Liver, FXN, Cardiac muscle, Investment, BAG3, Pharmaceutical industry, Management, Vaccine, Agriculture, Hospital

CHARLESTOWN, Mass., Jan. 10, 2023 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing genetic medicines for neuromuscular and cardiac diseases, today outlined its strategic priorities for 2023. These announcements are being made in advance of Solid’s presentation at the 41st Annual J.P. Morgan Healthcare Conference scheduled for January 12 at 9:00 am PT.

Key Points: 
  • “The Solid leadership team has deep expertise in precision genetic medicine, and our diversified pipeline targets diseases with high unmet need, strong scientific rationale, and significant commercial potential.
  • Solid remains focused on developing next-generation AAV capsid libraries with two strategies designed to enhance cardiac and skeletal muscle tropism.
  • Clear mechanistic rationale between genetic BAG3 insufficiency and myofilament damage, poor contraction and heart failure
    Solid’s construct candidates utilize the AAVrh74 capsid and cardiac specific promoter.
  • In early nonclinical studies, the specific capsid and cardiac-specific promoter combination increased cardiac expression while reducing expression in the liver.

Solid Biosciences Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Retrieved on: 
Monday, December 5, 2022
Acquisition, Ataxia, RSU, Employment, Science, Calendar, Disease management, Solid, Patient, BAG3, Duchenne

The grants were approved by the Company’s board of directors as an inducement material to each of Mr. Cumbo, Mr. Howton, Dr. Marlowe, Dr. Hanrahan and Mr. Herzich entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).

Key Points: 
  • The grants were approved by the Company’s board of directors as an inducement material to each of Mr. Cumbo, Mr. Howton, Dr. Marlowe, Dr. Hanrahan and Mr. Herzich entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).
  • Mr. Howton received an inducement grant with an option to purchase up to 104,410 shares of common stock as well as an RSU award of 52,205 shares of common stock.
  • The grants were approved by the compensation committee of the Company’s board of directors as an inducement material to such employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).
  • The options have a ten-year term and an exercise price of $6.77 per share, the closing price per share of Solid Biosciences’ common stock as reported by Nasdaq on December 2, 2022.

Solid Biosciences Announces Closing of Acquisition of AavantiBio and Concurrent $75 Million Private Placement

Retrieved on: 
Monday, December 5, 2022
Pura, FA, Acquisition, Duchenne muscular dystrophy, BofA Securities, Security (finance), Wilmer Cutler Pickering Hale and Dorr, FDA, NOS1, LLP, Safety, Science, Ataxia, Sidley Austin, Cardiomyopathy, Disease management, AAV, Investment, IND, Patient, BAG3, Vaccine, Pharmaceutical industry, Duchenne, Solid

CHARLESTOWN, Mass., Dec. 05, 2022 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced the closing of its acquisition of AavantiBio, a privately held gene therapy company focused on transforming the lives of patients with Friedreich’s ataxia and rare cardiomyopathies, including its pipeline assets and net cash. The combined company will focus on advancing a portfolio of neuromuscular and cardiac programs, including SGT-003, a differentiated gene transfer candidate, for the treatment of Duchenne, AVB-202, a gene transfer candidate for the treatment of Friedreich’s ataxia, AVB-401 for BAG3 mediated dilated cardiomyopathy, and additional assets for the treatment of undisclosed cardiac diseases. Bo Cumbo, the Chief Executive Officer of AavantiBio, will assume the role of President and CEO of Solid Biosciences.

Key Points: 
  • Bo Cumbo, the Chief Executive Officer of AavantiBio, will assume the role of President and CEO of Solid Biosciences.
  • Concurrent with the closing of the merger, Solid closed the previously announced $75 million private placement with a select group of institutional investors and accredited investors.
  • Solid’s stockholders approved the issuance of shares of Solid common stock in the transactions on December 1, 2022, along with the other proposals presented at the meeting.
  • Following the closing of the merger and private placement, Solid has total cash and investments of approximately $215 million.

Solid Biosciences Announces Stockholder Approval of Acquisition of AavantiBio

Retrieved on: 
Thursday, December 1, 2022
Solid, Family, Safety, Bangladesh Securities and Exchange Commission, Cardiomyopathy, FDA, Mutation, Security (finance), Patient, Disease, Acquisition, BAG3, Tripura Merger Agreement, Duchenne muscular dystrophy, Risk management, Duchenne

CHARLESTOWN, Mass., Dec. 01, 2022 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced the results of the special meeting of the stockholders held on December 1, 2022. At the special meeting, Solid’s stockholders voted in favor of all proposals, including the proposal to issue shares of Solid’s common stock to stockholders of AavantiBio in connection with the acquisition of AavantiBio pursuant to the agreement and plan of merger dated September 29, 2022 (the “Merger Agreement”) and to certain institutional investors in connection with a concurrent $75 million private placement. AavantiBio, Inc. is a privately held gene therapy company focused on transforming the lives of patients with Friedreich’s ataxia and rare cardiomyopathies.

Key Points: 
  • AavantiBio, Inc. is a privately held gene therapy company focused on transforming the lives of patients with Friedreichs ataxia and rare cardiomyopathies.
  • The combined company will focus on advancing a portfolio of neuromuscular and cardiac programs, led by SGT-003, a differentiated gene transfer candidate, for the treatment of Duchenne.
  • Solid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne.
  • However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.