Fabry disease

Sangamo Therapeutics Reports Recent Business Highlights and Second Quarter 2023 Financial Results

Retrieved on: 
Martedì, Agosto 8, 2023
Biotechnology, Pharmaceutical, Genetics, Health, Pfizer, Central nervous system, Therapy, Gene, Haemophilia A, BLA, Biogen, Safety, Fabry disease, Phytoene synthase, Research, Brain, AAV, Science, Fast track (FDA), U.S. FDA, Sodium, Phase 3, Nav1.7, Novartis, Patient, Prion, Primate, Annual general meeting, Cell, American Society for Cell Biology, Eli Lilly and Company, MAA, FDA, STAAR, Enzyme replacement therapy, IND, Neurology, Haemophilia, Society, Pharmaceutical industry, Medical device, Vaccine, Fabry, Chroma, Sanofi, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and second quarter 2023 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and second quarter 2023 financial results.
  • We are pleased to have executed several business development deals, demonstrating the excitement in Sangamo’s science and platform,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • Revenues for the second quarter ended June 30, 2023 were $6.8 million, compared to $29.4 million for the same period in 2022.
  • Additionally, revenues relating to our collaboration agreement with Kite decreased by $5.1 million, reflecting a reduction in collaboration activities during the quarter.

Protalix BioTherapeutics Reports Second Quarter 2023 Financial and Business Results

Retrieved on: 
Lunedì, Agosto 7, 2023
Protalix BioTherapeutics, Medsafe, European Medicines Agency, Tax, MD, Patient, European, Fabry disease, BLA, NYSE, Calendar, Gout, Half-life, Pharmacokinetics, Research, EMA, Russell, FIH, Growth, FDA, PEGylation, Biologics license application, Safety, Civil service commission, Health, Baylor University Medical Center, Israel Innovation Authority, FASN, Webcast, MAA, HDEC, European Commission, KOL, Clinical trial, EDT, Marketing, ERT, EC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Vaccine, Pharmaceutical industry, Drug, Life insurance, Online shopping, EU, Fabry, Chiesi Farmaceutici

CARMIEL, Israel, Aug. 7, 2023 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, today reported financial results for the second quarter ended June 30, 2023 and provided a business update on recent regulatory, clinical and corporate developments.

Key Points: 
  • "2023 has been a transformational year for Protalix thus far," said Dror Bashan, Protalix's President and Chief Executive Officer.
  • On May 5, 2023, the European Commission (EC) granted marketing authorization to Elfabrio (pegunigalsidase alfa) in the European Union.
  • On June 26, 2023, the Company was included in the broad-market Russell 3000® Index at the conclusion of the 2023 Russell indexes annual reconstitution.
  • Financial expenses, net were $0.8 million for the three months ended June 30, 2023, compared to financial income, net of $0.2 million for the three months ended June 30, 2022.

uniQure Announces Second Quarter 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Martedì, Agosto 1, 2023
Senior, Takeda, Abbott Laboratories, CSL, American Society of Gene and Cell Therapy, Associate, Research and development, Development, Patient, Cell, Toxicology, Fabry disease, ALS, Gene, General counsel, Research, Forma, SOD1, Corporation, CSF, Wells Fargo, IND, Unified, Conference, Safety, GLP, Abstract, Natural history, CSL Behring, Cancer, AAV, Primate, Therapy, Takeda Pharmaceutical Company, Commercialization, AFL, Clinical trial, Sale, Novartis, Takeda Oncology, Novo Nordisk, Immunosuppression, Society, Pharmaceutical industry, Cryptocurrency, EU, Pfizer

and AMSTERDAM, Aug. 01, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the second quarter of 2023 and highlighted recent progress across its business.

Key Points: 
  • and AMSTERDAM, Aug. 01, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the second quarter of 2023 and highlighted recent progress across its business.
  • The Company continues to make progress enrolling the second, high-dose cohort of nine patients and expects to complete enrollment in the third quarter of 2023.
  • The Company expects to initiate a Phase I/II clinical study of AMT-162 in the second half of 2023.
  • The Company expects cash, cash equivalents and investment securities will fund operations into the second quarter of 2026.

Latest Updates of 7 Viva's Portfolio Companies

Retrieved on: 
Venerdì, Luglio 28, 2023
Dai, FALCON, Head, Acquisition, Patient, Fabry disease, SUA, Tissue, Pharma, Gout, Therapy, Heart failure, CSO, GABAA receptor negative allosteric modulator, Research, NAM, VBI, Small RNA, Medicine, Liver, Growth, CIO, Listeria monocytogenes non-coding RNA, Phase, FDA, URAT1, G protein-coupled receptor, SRT, PAR2, DSM-IV codes, Gq alpha subunit, Glaucoma, Ocular hypertension, SAN, Mercury (element), Allosteric regulation, IOP, Flare, Central nervous system, Drug development, Quality of life, Facet joint arthrosis, RNA, Novartis, Cardiology, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, Fine chemical, Riparian zone, Domain, Pfizer

WATERTOWN, Mass.-- Riparian Pharmaceuticals, a Viva Biotech portfolio company, is a biotechnology company focused on discovering novel therapeutics for cardiovascular diseases. Riparian today announced it has entered into an exclusive license agreement and research agreement with Pfizer.

Key Points: 
  • WATERTOWN, Mass.-- Riparian Pharmaceuticals, a Viva Biotech portfolio company, is a biotechnology company focused on discovering novel therapeutics for cardiovascular diseases.
  • Riparian today announced it has entered into an exclusive license agreement and research agreement with Pfizer.
  • In exchange for exclusive rights to a Riparian preclinical program, Pfizer will make upfront and milestone payments, as well as pay royalties on sales of resulting therapeutics.
  • DTx Pharma utilizes its groundbreaking FALCON™ platform, a conjugated oligonucleotide technology using fatty acid ligands, to tackle the delivery challenges of oligonucleotide therapies.

AceLink Therapeutics Receives FDA Clearance to Initiate a Phase 2 Study of AL1211 in Patients with Fabry Disease

Retrieved on: 
Mercoledì, Giugno 21, 2023
Health, FDA, General Health, Clinical Trials, Research, Science, Pharmaceutical, Male, Fabry disease, Quality of life, Plasma, ERT, Patient, GCS, Enzyme replacement therapy, Therapy, Strong, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Safety, Pharmaceutical industry

“FDA clearance for AL1211 marks an important clinical milestone for AceLink,” said Jerry Shen, Ph.D., Chief Executive Officer and Founder of AceLink Therapeutics.

Key Points: 
  • “FDA clearance for AL1211 marks an important clinical milestone for AceLink,” said Jerry Shen, Ph.D., Chief Executive Officer and Founder of AceLink Therapeutics.
  • Phase 1 clinical trials evaluated both single ascending dosing and multiple ascending dosing.
  • AL1211 was orally administered to healthy volunteers as a single dose up to 60 mg or multiple doses of up to 30 mg once daily for 14 days.
  • Globotriasosylceramide (Gb3), the disease-causing lipid of Fabry Disease, was also significantly reduced.

Protalix BioTherapeutics to Host In-Person KOL Breakfast following the Approval of Elfabrio® in both the United States and the European Union for Adults with Fabry Disease

Retrieved on: 
Giovedì, Giugno 8, 2023
Breakfast, KOL, Protalix BioTherapeutics, EDT, Baylor University Medical Center, Patient, FASN, Fabry disease, FDA, NYSE, Chiesi Farmaceutici

CARMIEL, Israel, June 8, 2023 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, today announced it will host an in-person KOL breakfast in New York on Tuesday, June 27, 2023 at 8:00 a.m. Eastern Daylight Time (EDT), with presentations beginning at 8:30 a.m. EDT.

Key Points: 
  • The event will feature a discussion of the recent approval by the U.S. Food and Drug Administration (FDA) of Elfabrio (pegunigalsidase alfa-iwxj) in the United States for the treatment of adult patients with Fabry disease.
  • Ankit Mehta, M.D., FASN (Baylor University Medical Center) will discuss the opportunity for Elfabrio to address the unmet needs in Fabry disease; Giacomo Chiesi, Head of Chiesi Global Rare Diseases, the Company's commercial partner, will discuss Chiesi's commercial capabilities; and the Company's leadership will provide insight into the Company's strategy and future plans.
  • A live question and answer session will follow the presentations.
  • Registration for the event is available at https://lifescievents.com/event/protalix/ , or by clicking here .

Carina Biotech Appoints Two New U.S. Based Biotechnology Executives to the Board of Directors

Retrieved on: 
Lunedì, Giugno 5, 2023
CLL, Gaucher's disease, Medicine, University, Chronic lymphocytic leukemia, SLL, Acute lymphoblastic leukemia, Biotechnology, Food, Food and Drug Administration, MBA, Providence College, University of Bern, Pharmacyclics, Audit committee, Waldenström macroglobulinemia, Genzyme, Patient, Mantle cell lymphoma, Hyperphosphatemia, Cero, Bone marrow, Kite Pharma, Fabry disease, FDA, Therapy, Suffolk University, Imiglucerase, IND, Sevelamer, Safety, Pharmaceutical industry, Ibrutinib

ADELAIDE, Australia, June 05, 2023 (GLOBE NEWSWIRE) -- Carina Biotech (Carina), a cell therapy immuno-oncology company, today announced the appointment of Michael S. Wyzga, M.B.A., and Remus Vezan M.D., Ph.D., to its Board of Directors.

Key Points: 
  • ADELAIDE, Australia, June 05, 2023 (GLOBE NEWSWIRE) -- Carina Biotech (Carina), a cell therapy immuno-oncology company, today announced the appointment of Michael S. Wyzga, M.B.A., and Remus Vezan M.D., Ph.D., to its Board of Directors.
  • Mr. Wyzga is an accomplished biotechnology executive who brings to Carina’s Board over two decades of expertise as a leader and advisor to life sciences companies.
  • “We are delighted to welcome two seasoned executives that bring a wealth of expertise in the U.S. biotechnology industry as independent Directors to the Carina Board,” remarked Leanna Read, Ph.D., Chair of Carina’s Board of Directors.
  • Their insight and contribution have been invaluable.”
    Mr. Gosling will continue to support Carina on the Governance, People and Remuneration Committee, whilst Mr. Latham will remain Company Secretary.

Protalix BioTherapeutics set to join Russell 3000® Index

Retrieved on: 
Martedì, Maggio 23, 2023
Growth, FTSE Russell, Fabry disease, Russell, NYSE, Protalix BioTherapeutics, Cryptocurrency

Membership in the US all-cap Russell 3000® Index, which remains in place for one year, means automatic inclusion in the large-cap Russell 1000® Index or small-cap Russell 2000® Index as well as the appropriate growth and value style indexes.

Key Points: 
  • Membership in the US all-cap Russell 3000® Index, which remains in place for one year, means automatic inclusion in the large-cap Russell 1000® Index or small-cap Russell 2000® Index as well as the appropriate growth and value style indexes.
  • FTSE Russell determines membership for its Russell indexes primarily by objective, market-capitalization rankings and style attributes.
  • Russell indexes are part of FTSE Russell, a leading global index provider.
  • For more information on the Russell 3000® Index and the Russell indexes reconstitution, go to the "Russell Reconstitution" section on the FTSE Russell website .

Sangamo Therapeutics Receives U.S. FDA Fast Track Designation for Isaralgagene Civaparvovec for the Treatment of Fabry Disease

Retrieved on: 
Lunedì, Maggio 22, 2023
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Genetics, Clinical Trials, Priority review, Southwest General Health Center, Fabry disease, Therapy, STAAR, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Patient, Food, Accelerated approval (FDA), Pharmaceutical industry, Fabry, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease.
  • Companies granted this designation are given the opportunity for more frequent interactions with the FDA.
  • “We are thrilled with the FDA’s decision to grant Fast Track Designation for ST-920.
  • Fabry is a debilitating disease with life-long impact,” said Nathalie Dubois-Stringfellow, Ph.D, Sangamo’s Senior Vice President, Chief Development Officer.

Protalix BioTherapeutics Announces $20 Million Milestone Payment from Chiesi Global Rare Diseases

Retrieved on: 
Giovedì, Maggio 18, 2023
Protalix BioTherapeutics, License, Patient, Fabry disease, FDA, NYSE, Pharmaceutical industry, Chiesi Farmaceutici

CARMIEL, Israel, May 18, 2023 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, today announced that it is eligible to receive a $20 million milestone payment from its commercial partner, Chiesi Global Rare Diseases, a business unit of the Chiesi Group. The milestone payment was triggered by the approval by the U.S. Food and Drug Administration (FDA) of ELFABRIO (pegunigalsidase alfa-iwxj) for the treatment of adult patients with Fabry disease, which was announced on May 10, 2023, and is payable within 30 days of the FDA approval date.

Key Points: 
  • CARMIEL, Israel, May 18, 2023 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, today announced that it is eligible to receive a $20 million milestone payment from its commercial partner, Chiesi Global Rare Diseases, a business unit of the Chiesi Group.
  • "Receiving this milestone payment resulting from the FDA's approval of ELFABRIO is an important step for Protalix as it further to strengthens our financial position," said Dror Bashan, Protalix's President and Chief Executive Officer.
  • "We continue to be grateful to our commercial partner, Chiesi, who has the global expertise to maximize the market potential of pegunigalsidase alfa."
  • On a proforma basis, including the receipt of the $20.0 million milestone payment, cash and cash equivalents as of May 18, 2023 is $51.6 million.