BLA

Carmell to Host Investor Webinar, “Unveiling the Secretome: On the Eve of Product Launch” Featuring Key Opinion Leaders in the Medical Aesthetics Field

Retrieved on: 
Monday, March 18, 2024
Harvard Medical School, Worldwide, Research, Doctor of Philosophy, Protein, Regius Professor, Skin, Secretome, Safety, FACS, FRCS, BLA, Peptide, Vaccine

Carmell's Secretome technology delivers 1000+ growth factors, proteins and peptides extracted from allogeneic human platelets in a twelve-month shelf-stable and biologically active formulation.

Key Points: 
  • Carmell's Secretome technology delivers 1000+ growth factors, proteins and peptides extracted from allogeneic human platelets in a twelve-month shelf-stable and biologically active formulation.
  • Carmell’s technology was previously being developed as a BLA and has Phase 2 human clinical data supporting safety and efficacy.
  • In comparison, other aesthetic products feature 1-2 synthetic proteins with sparse evidence of clinical efficacy.
  • Carmell aims to launch a limited-edition product this month followed by 9 other skincare products over the summer 2024.

Abeona Therapeutics Reports Full Year 2023 Financial Results and Announces Completion of FDA Inspections

Retrieved on: 
Monday, March 18, 2024
Research, AAV, Advisory Committee, Form FDA 483, PLI, Sale, EIR, University, Trial of the century, Rett syndrome, Concha bullosa, Priority review, Investment, IP, Therapy, Safety, REMS, EB, Food, FDA, BLA, RDEB, PDUFA, Pharmaceutical industry

On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit.

Key Points: 
  • On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit.
  • In January 2024, the FDA completed both a Bioresearch Monitoring (BIMO) inspection of Abeona and the Mid-Cycle Meeting regarding the pz-cel BLA.
  • Following completion of the BIMO inspection, the FDA inspector did not issue any observations or FDA Form 483s.
  • In March 2024, Abeona received a written Establishment Inspection Report (EIR), the formal report from the FDA regarding the BIMO inspection.

Larimar Therapeutics Reports Fourth Quarter and Full Year 2023 Operating and Financial Results and Provides Update on Nomlabofusp Development

Retrieved on: 
Thursday, March 14, 2024
Research, Database, Gene expression, Disease, Patient, Accounting, OLE, Insurance, Ataxia, Clinical trial, Skin, Therapy, Safety, Pharmacokinetics, Interim, FA, FDA, BLA, Pharmaceutical industry

BALA CYNWYD, Pa., March 14, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today reported its fourth quarter and full year 2023 operating and financial results.

Key Points: 
  • BALA CYNWYD, Pa., March 14, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today reported its fourth quarter and full year 2023 operating and financial results.
  • Nomlabofusp was generally well-tolerated and demonstrated dose-dependent increases in skin and buccal cell frataxin levels.
  • Research and development expenses for the fourth quarter of 2023 were $10.6 million compared to $7.2 million for the fourth quarter of 2022.
  • General and administrative expenses for the fourth quarter of 2023 were $3.5 million compared to $3.2 million for the fourth quarter of 2022.

Autolus Therapeutics Reports Full Year 2023 Financial Results and Business Updates

Retrieved on: 
Thursday, March 14, 2024
Nucleus, Bone marrow, BCMA, BioNTech, B-ALL, Patient, ALL, SLE, Engineering, Minocycline, European Medicines Agency, Therapy, Research, Canadian Aviation Regulations, Flow cytometry, ASH, General counsel, Immunotherapy, SVP, EMA, Legal Affairs, Trial of the century, GMP, SME, BLA, Nature Communications, Development, UCL, Biologics license application, Multiple myeloma, MIA, University College London, Senior, TRBC, GD2, ASCO, FDA, CD19, Conference call, Conference, MAA, EHA, ADS, MHRA, T cell, ACS Chemical Biology, PDUFA, Autoimmune disease, Pharmaceutical industry

“For now, we remain fully focused on preparing for a potential obe-cel launch and successfully transitioning Autolus to a commercial stage company.

Key Points: 
  • “For now, we remain fully focused on preparing for a potential obe-cel launch and successfully transitioning Autolus to a commercial stage company.
  • In February 2024, Autolus promoted Dr. Chris Williams to Chief Business Officer and Alex Driggs to Senior Vice President, Legal Affairs and General Counsel.
  • Dr. Leiderman brings extensive transactional and financial expertise, and Mr. Azelby brings more than 30 years of biopharmaceutical leadership and commercial experience to Autolus’ Board.
  • Financial Results for the Year Ended December 31, 2023

aTyr Pharma Announces Fourth Quarter and Full Year 2023 Results and Provides Corporate Update

Retrieved on: 
Thursday, March 14, 2024
PDT, Research, Conference, Interest, ATS, Biologics license application, Patient, SAN, Howard University, Knowledge, EAP, ATyr Pharma, EDT, NRP2, American Thoracic Society, Downstream, Investment, Clinical trial, Small RNA, Neuropilin, Myelocyte, Safety, Sarcoidosis, Immune system, FGF2, FGFR4, BLA, Pharmaceutical industry

SAN DIEGO, March 14, 2024 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE) (“aTyr” or the “Company”), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced fourth quarter and full year 2023 results and provided a corporate update.

Key Points: 
  • SAN DIEGO, March 14, 2024 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE) (“aTyr” or the “Company”), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced fourth quarter and full year 2023 results and provided a corporate update.
  • Based on current enrollment projections, the Company anticipates completing enrollment in the study in the second quarter of 2024.
  • Interested parties may access the call by registering here in order to obtain a dial in, personalized passcode and webcast information.
  • Links to a live audio webcast and replay may be accessed on the aTyr website Events page at: http://investors.atyrpharma.com/events-and-webcasts .

Pacira BioSciences Announces PCRX-201 Granted Regenerative Medicine Advance Therapy (RMAT) Designation for the Treatment of Osteoarthritis of the Knee

Retrieved on: 
Wednesday, March 13, 2024
Knee, Congress, Biologics license application, Patient, ATMP, Research, World Congress, OARSI, European Medicines Agency, WOMAC, Osteoarthritis Research Society International, KOOS, Drug development, Safety, Pain, Vasopressin receptor 2, DVD-Audio, Food, Osteoarthritis, FDA, BLA, Pharmaceutical industry

TAMPA, Fla., March 13, 2024 (GLOBE NEWSWIRE) -- Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to PCRX-201 (enekinragene inzadenovec), the company’s novel, intra-articular helper-dependent adenovirus (HDAd) gene therapy product candidate that codes for interleukin-1 receptor antagonist (IL-1Ra), for the treatment of osteoarthritis of the knee.

Key Points: 
  • PCRX-201 was well tolerated, with efficacy observed through at least 52 weeks at all doses and cohorts.
  • Preliminary 36-week data were presented at the Osteoarthritis Research Society International (OARSI) 2023 World Congress, the premier annual international forum in osteoarthritis research and treatment.
  • The 52-week data have been accepted for presentation at OARSI 2024 taking place in Vienna, Austria in April 2024 and the company expects to present 104-week efficacy and safety data later this year.
  • PCRX-201 was also granted Advanced Therapy Medicinal Products (ATMP) designation by the European Medicines Agency in May 2023.

Faron’s Financial Statement Release January 1 to December 31, 2023

Retrieved on: 
Wednesday, March 13, 2024
Head, Q&A, Survival, Immunotherapy, Patient, Medicine, Standard of care, CMML, Macrophage, Faron Pharmaceuticals, Neoplasm, EET, Rheumatology, PD-1, TME, PR, ASH, AML, Cancer, Johnson & Johnson, Safety, Azacitidine, MDS, BLA, Decitabine, Food, ODD, Nasdaq First North, PD, Tumor microenvironment, Manuscript, CRS, SICAV, MD, Interferon beta-1a, Haematopoietic system, GMT, FDA, AIM, Neurotoxicity, Infection, EUR, Sanofi, SOC, Metabolism, European Hematology Association, EDT, MBA, IPF, Facility, Treasury, Society, Cytokine release syndrome, CPA, Pharmaceutical industry

As at March 13, 2024, the Company is in compliance with all IPF financial covenants as agreed with the waiver letter.

Key Points: 
  • As at March 13, 2024, the Company is in compliance with all IPF financial covenants as agreed with the waiver letter.
  • Loss for the period for the financial year ended December 31, 2023, was EUR 30,9 million (2022: EUR 28,7 million).
  • In June 2023, Faron conducted a placement of 2,601,510 newly issued treasury shares to investors to raise EUR 6,6 million gross.
  • In October 2023, the Company successfully raised EUR 7,1 million gross through the issuance of 2,491,998 ordinary shares to investors.

Shoals Technologies Group Asserts Its Intellectual Property Rights and Vows to Defend Patent

Retrieved on: 
Tuesday, March 12, 2024
Patent, Shoal, Voltage, ITC, BLA

11,689,153 (the “‘153 patent”) related to Shoals’ Big Lead Assembly (“BLA”) technology.

Key Points: 
  • 11,689,153 (the “‘153 patent”) related to Shoals’ Big Lead Assembly (“BLA”) technology.
  • The ‘153 patent is one of Shoals’ strongest patents as evidenced by the ITC’s claim construction ruling, and the ITC’s recent ruling related to the ‘739 patent does not affect Shoals’ case against Voltage related to the ‘153 patent.
  • “Both the ‘739 and ‘153 patents remain active and valid,” said Brandon Moss, CEO of Shoals Technologies Group.
  • Shoals has also recently received Notices of Allowance for two patent applications, U.S. Patent Application Nos.

Global Cell and Gene Therapy Markets Report 2024-2028: Focus on Zolgensma, Yescarta, Kymriah, Abecma, Maci, Tecartus, Epicel - ResearchAndMarkets.com

Retrieved on: 
Wednesday, April 3, 2024
Health, Genetics, Growth, PTC Therapeutics, Prevalence, Gene editing, ESG, CSL Behring, Roche, Etranacogene dezaparvovec, Gene, Gilead Sciences, Cancer, Eladocagene exuparvovec, Therapy, CRISPR, Mallinckrodt, PTC, Probability, BLA, Pharmaceutical industry

The "Global Cell and Gene Therapy Market" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Cell and Gene Therapy Market" report has been added to ResearchAndMarkets.com's offering.
  • The Global Cell and Gene Therapy Market is expected to grow at a compound annual growth rate (CAGR) of 26.40% from 2023 through 2028.
  • The scope of this study encompasses an investigation of the cell and gene therapy market.
  • The report analyzes cell and gene therapy market based on therapy type, product, application, and region.

Rocket Pharmaceuticals Announces European Medicines Agency Acceptance of RP-L102 Marketing Authorization Application for the Treatment of Fanconi Anemia

Retrieved on: 
Tuesday, April 2, 2024
Biotechnology, Pharmaceutical, Oncology, General Health, Health, Genetics, Clinical Trials, Other Health, Transplant, DNA repair, Medication, Biologics license application, MAA, Risk, FANCA, Patient, Fanconi anemia, Pharming, DNA, BMF, HSCT, EMA, Bone marrow failure, Death, Birth, Life, MBA, European Medicines Agency, Marketing, FA, Disorder, FDA, BLA, Genetic disorder, Food

“The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder.

Key Points: 
  • “The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder.
  • Currently, there are no existing options to potentially prevent BMF for patients with FA,” said Kinnari Patel, Pharm.D., MBA, President, Head of R&D and Chief Operating Officer, Rocket Pharma.
  • The safety profile was highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, was well tolerated.
  • The Biologics License Application (BLA) for FA remains on track for submission to the U.S. Food and Drug Administration (FDA) in the first half of 2024.