Ultragenyx

North River Leerink’s Successful 100 Chestnut Street Development Continues Somerville’s Growth as a Leading Life Science and Innovation Hub

Retrieved on: 
Thursday, February 29, 2024

At the heart of this transformation stands 100 Chestnut, a purpose-built 200,000 sq.

Key Points: 
  • At the heart of this transformation stands 100 Chestnut, a purpose-built 200,000 sq.
  • ft. Life Science building that is 90% leased, reflecting NRL's passion and focus on developing world-class buildings that enable innovation and support the creation of sustainable and vibrant communities.
  • Our goal is to contribute towards the development of a rich, vibrant and diverse district focused on fostering Art and enabling innovation in the Life Science/Biotech industry,” said Jeff Leerink, Managing Partner at NRL.
  • And most recently, ADA Forsyth, a leading institute dedicated to improving oral and overall health through innovative research and community outreach, has leased 76,000 sq.

Ultragenyx Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Update

Retrieved on: 
Thursday, February 15, 2024

NOVATO, Calif., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2023 and reaffirmed its financial guidance for 2024.

Key Points: 
  • Total operating expenses for the fourth quarter of 2023 were $249 million, including non-cash stock-based compensation of $34 million.
  • Total operating expenses for the year ended December 31, 2023 were $1,003 million, including $135 million of non-cash stock-based compensation.
  • For the fourth quarter of 2023, Ultragenyx reported net loss of $123 million, or $1.52 per share basic and diluted, compared with a net loss for the fourth quarter of 2022 of $152 million, or $2.16 per share, basic and diluted.
  • ET to discuss the fourth quarter and full year 2023 financial results and provide a corporate update.

Disc Medicine Strengthens Leadership Team with Appointment of Seasoned Industry Executive Jean Franchi as Chief Financial Officer, and Promotion of Jonathan Yu to Chief Operating Officer

Retrieved on: 
Wednesday, February 7, 2024

Ms. Franchi will be responsible for finance, investor relations, and other functions and will lead the company’s capital formation and financial strategy.

Key Points: 
  • Ms. Franchi will be responsible for finance, investor relations, and other functions and will lead the company’s capital formation and financial strategy.
  • “We are excited to welcome an executive of Jean’s caliber and depth of experience to the Disc team, and I’m delighted to announce the promotion of a key executive, Jonathan Yu, to Chief Operating Officer,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc.
  • The team has built a unique company with a strong foundation and pipeline with enormous potential,” said Ms. Franchi.
  • Prior to Replimune, Ms. Franchi served as Chief Financial Officer for Merrimack Pharmaceuticals, Dimension Therapeutics, which was acquired by Ultragenyx, and Good Start Genetics.

Ultragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza® ▼ (evinacumab) for Adolescents and Adults Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)

Retrieved on: 
Thursday, January 4, 2024

Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH).

Key Points: 
  • Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH).
  • Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for this rare and debilitating condition.
  • "We are very pleased that NICE has approved the use of evinacumab within the NHS.
  • The NICE committee acknowledged that clinical trial evidence shows that Evkeeza, combined with other lipid-lowering therapies (LLTs), can lower LDL-C levels when statins and other LLTs have not reduced them enough.

Amylyx Pharmaceuticals Appoints Camille L. Bedrosian, MD, as Chief Medical Officer

Retrieved on: 
Tuesday, November 28, 2023

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the appointment of Camille L. Bedrosian, MD, as Chief Medical Officer (“CMO”).

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the appointment of Camille L. Bedrosian, MD, as Chief Medical Officer (“CMO”).
  • In this role, Dr. Bedrosian will lead global functions in medical affairs, regulatory, safety and pharmacovigilance, biometrics, clinical development, and clinical operations.
  • I’m excited to join Amylyx and contribute to bringing innovative, new treatment options to individuals and communities suffering from neurodegenerative diseases.
  • “We also want to thank our longtime Chief Medical Officer, Dr. Patrick Yeramian, for his many years of dedication and commitment to building Amylyx into the successful company it is today.

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

Retrieved on: 
Monday, December 18, 2023

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

Key Points: 
  • COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).
  • Andelyn has successfully manufactured GMP supply of UX111 for patient dosing and will be performing the PPQ batches necessary for any future regulatory filings.
  • Andelyn is honored to continue its close partnership to advance the UX111 Program to bring treatment to MPS IIIA patients sooner."
  • With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases.

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

Retrieved on: 
Monday, December 18, 2023

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

Key Points: 
  • COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).
  • Andelyn has successfully manufactured GMP supply of UX111 for patient dosing and will be performing the PPQ batches necessary for any future regulatory filings.
  • Andelyn is honored to continue its close partnership to advance the UX111 Program to bring treatment to MPS IIIA patients sooner."
  • With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases.

Ultragenyx Reports Third Quarter 2023 Financial Results and Corporate Update

Retrieved on: 
Thursday, November 2, 2023

NOVATO, Calif., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter ended September 30, 2023 and provided its financial guidance for the year.

Key Points: 
  • For the full year 2023, the company expects:
    Crysvita revenue in the range of $325 million to $340 million.
  • This includes all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023.
  • In May 2023, Ultragenyx announced the last patient had been dosed in the Phase 3 study.
  • ET to discuss the third quarter 2023 financial results and provide a corporate update.

TATAA Announces Formation of Scientific Advisory Board

Retrieved on: 
Tuesday, October 31, 2023

GOTHENBURG, Sweden, Oct. 31, 2023 /PRNewswire/ -- TATAA Biocenter (the "Company" or "TATAA"), a leading global CRO for high-quality molecular analysis, announced the formation of its Scientific Advisory Board ("SAB").

Key Points: 
  • GOTHENBURG, Sweden, Oct. 31, 2023 /PRNewswire/ -- TATAA Biocenter (the "Company" or "TATAA"), a leading global CRO for high-quality molecular analysis, announced the formation of its Scientific Advisory Board ("SAB").
  • "We are excited to have such a distinguished group of experts on our Scientific Advisory Board.
  • The Scientific Advisory Board demonstrates our commitment to delivering innovative solutions and exceptional services to our clients, and we look forward to working closely with them to achieve our goals."
  • said Dr. Sofia Adolfsson, Scientific Officer and Head of Bioinformatics at TATAA.

TATAA Announces Formation of Scientific Advisory Board

Retrieved on: 
Tuesday, October 31, 2023

GOTHENBURG, Sweden, Oct. 31, 2023 /PRNewswire/ -- TATAA Biocenter (the "Company" or "TATAA"), a leading global CRO for high-quality molecular analysis, announced the formation of its Scientific Advisory Board ("SAB").

Key Points: 
  • GOTHENBURG, Sweden, Oct. 31, 2023 /PRNewswire/ -- TATAA Biocenter (the "Company" or "TATAA"), a leading global CRO for high-quality molecular analysis, announced the formation of its Scientific Advisory Board ("SAB").
  • "We are excited to have such a distinguished group of experts on our Scientific Advisory Board.
  • The Scientific Advisory Board demonstrates our commitment to delivering innovative solutions and exceptional services to our clients, and we look forward to working closely with them to achieve our goals."
  • said Dr. Sofia Adolfsson, Scientific Officer and Head of Bioinformatics at TATAA.