Glycogen storage disease type II

Evox Therapeutics Presents Progress Across its DeliverEX™ Exosome Therapeutics Platform with Nine Presentations at the International Society for Extracellular Vesicles (ISEV) Annual Meeting

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Thursday, May 26, 2022
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In parallel, we are also focused on advances in exosome manufacturing, including exploring new ways to produce exosome therapeutics.

Key Points: 
  • In parallel, we are also focused on advances in exosome manufacturing, including exploring new ways to produce exosome therapeutics.
  • We believe our advances will allow exosome therapeutics access to cellular compartments and tissues that are currently out of reach using other approaches.
  • This could provide significant benefit for both creating feasible dosing regimens and to ensure sufficient plasma residence time for tissue-specific targeting.
  • Engineered display of ligands on the surface of exosomes can be used to increase specific targeting of exosomes to desired tissues.

AskBio President of Therapeutics Dr. Katherine High receives Award from American Society of Gene and Cell Therapy

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Monday, May 23, 2022
NAS, Physician, Muscular dystrophy, Patent, Parkinson's disease, Hospital, Capsid, Food, National, Multimedia, Patient, Goal, Spark Therapeutics, Association, Therapy, Clinical trial, Society, Bayer, National academy, COVID-19, RESEARCH, Spinal muscular atrophy, Trust, Feedback, Secondary research, Research, Gene, FDA, Glycogen storage disease type II, PARK, Food and Drug Administration, Heart, Disagreement, CHOP, AAV, Lists of diseases, Center, Kaay Raav Tumhi, Pompe, Human, Central nervous system, Technology, Inauguration, Research Triangle Park, Child, United States Patent and Trademark Office, Ageing, Voretigene neparvovec, Group, American Society of Gene and Cell Therapy, LinkedIn, Person, Cell, Growth, Mendell, MD, Medical device, Pharmaceutical industry

RESEARCH TRIANGLE PARK, N.C.  , May 23, 2022 /PRNewswire/ --  Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that Dr. Katherine High, President of Therapeutics, received the Jerry Mendell award for Translational Science from the American Society of Gene and Cell Therapy (ASGCT).  Recently inducted as a member of the National Academy of Sciences (NAS), Dr. High has received this award in recognition of her significant contributions to the field of genetic medicine.

Key Points: 
  • Today, at AskBio, Dr. High oversees a robust pipeline of gene therapeutics to treat various neuromuscular, central nervous system, cardiovascular and metabolic disorders, all at various stages of clinical development.
  • Today, the ASGCT is the largest association of individuals involved in gene and cell therapy research.
  • AskBio's gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library.
  • AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

AskBio to Present 11 Abstracts at Upcoming American Society of Gene and Cell Therapy's 25th Annual Meeting

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Monday, May 16, 2022
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RESEARCH TRIANGLE PARK, N.C., May 16, 2022 /PRNewswire/ -- Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that the Company will present 11 abstracts at the upcoming American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting being held May 16 – 19, 2022 at the Walter E. Washington Convention Center in Washington, D.C. 

Key Points: 
  • AskBio's gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library.
  • Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition.
  • Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements.
  • AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Codexis Presents Pre-Clinical Data Highlighting Gene Therapy Programs at the ASGCT 25th Annual Meeting

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Monday, May 16, 2022
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The pre-clinical data highlights enzyme variants engineered with Codexis CodeEvolver platform to offer potentially improved efficacy as compared to current enzymes when administered as transgenes in gene therapies for Hemophilia A, Fabry Disease, and Pompe Disease.

Key Points: 
  • The pre-clinical data highlights enzyme variants engineered with Codexis CodeEvolver platform to offer potentially improved efficacy as compared to current enzymes when administered as transgenes in gene therapies for Hemophilia A, Fabry Disease, and Pompe Disease.
  • Codexis used the CodeEvolver platform to identify a-galactosidase A (GLA) and a-glucosidase (GAA) variants to address Fabry Disease and Pompe Disease, respectively.
  • Codexis is a leading enzyme engineering company leveraging its proprietary CodeEvolverplatform to discover and develop novel, high performance enzymes and novel biotherapeutics.
  • Codexis expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law.

AVROBIO to Present at the H.C. Wainwright Global Investment Conference

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Monday, May 16, 2022
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(Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced members of its senior management team will be presenting virtually at the H.C. Wainwright Global Investment Conference, May 23-25, 2022.

Key Points: 
  • (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced members of its senior management team will be presenting virtually at the H.C. Wainwright Global Investment Conference, May 23-25, 2022.
  • The event will remain archived on the AVROBIO website for approximately 30 days.
  • AVROBIOs pipeline is powered by our industry-leading plato gene therapy platform, our foundation designed to deliver gene therapy worldwide.
  • AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Aro Biotherapeutics to Present New Preclinical Data Highlighting the Potential of Centyrin-siRNA Conjugates for the Treatment of Pompe Disease

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Wednesday, May 11, 2022
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Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced upcoming data presentations highlighting the therapeutic potential of its proprietary Centyrin-siRNA conjugate therapies for the treatment of Pompe disease.

Key Points: 
  • Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced upcoming data presentations highlighting the therapeutic potential of its proprietary Centyrin-siRNA conjugate therapies for the treatment of Pompe disease.
  • As a result of this mutation, patients with Pompe disease have elevated levels of glycogen which drive disease progression.
  • While treatment options exist in Pompe disease, they are limited and there is still great unmet need for the individual facing this disease.
  • These data represent a significant milestone for Aro, said Susan Dillon, PhD, Co-Founder and CEO of Aro Biotherapeutics.

Amicus Therapeutics Receives Notification of PDUFA Date Extensions for AT-GAA

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Tuesday, May 10, 2022
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The revised PDUFA action dates for miglustat and cipaglucosidase alfa are August 29, 2022 and October 29, 2022, respectively.

Key Points: 
  • The revised PDUFA action dates for miglustat and cipaglucosidase alfa are August 29, 2022 and October 29, 2022, respectively.
  • The FDA extended the PDUFA dates to allow additional time to review information submitted by the Company as part of its ongoing reviews.
  • We continue to work collaboratively with the FDA as it completes its review of the AT-GAA applications, said John F. Crowley, Chairman and Chief Executive Officer at Amicus Therapeutics.
  • You are cautioned not to place undue reliance on these forward-looking statements, which speak only of the date hereof.

AVROBIO Reports First Quarter 2022 Financial Results and Provides Business Update

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Tuesday, May 10, 2022
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(Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today reported financial results for the first quarter ended March 31, 2022 and provided a business update.

Key Points: 
  • (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today reported financial results for the first quarter ended March 31, 2022 and provided a business update.
  • Research and development expenses were $19.3 million for the first quarter of 2022 as compared to $18.5 million for the comparable period in 2021.
  • General and administrative expenses were $10.2 million for the first quarter of 2022 as compared to $8.4 million for the comparable period in 2021.
  • AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Baebies Partners with Medical Horizons to Bring SEEKER Newborn Screening Platform to Italy

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Tuesday, May 10, 2022
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FLORENCE, Italy, May 10, 2022 /PRNewswire/ -- Baebies, a growth-stage medical device company developing innovative products to enable early disease detection and multifunctional diagnostic testing for children and adults., in partnership with medical device supplier Medical Horizons, today announced newborns in Italy are being screened with the Baebies SEEKER platform.

Key Points: 
  • FLORENCE, Italy, May 10, 2022 /PRNewswire/ -- Baebies, a growth-stage medical device company developing innovative products to enable early disease detection and multifunctional diagnostic testing for children and adults., in partnership with medical device supplier Medical Horizons, today announced newborns in Italy are being screened with the Baebies SEEKER platform.
  • Powered by digital microfluidics technology, SEEKER is an FDA-cleared and CE-marked newborn screening platform for LSDs.
  • Dr. la Marca is a recognized global authority in newborn screening, who works closely with the International Society for Neonatal Screening to advance research and adoption of newborn screening around the world.
  • However, an amendment to current newborn screening legislation could expedite inclusion of new conditions like LSDs in screening panels around the country.

AVROBIO Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

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Friday, May 6, 2022
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In addition, the company has granted an aggregate of 67,177 restricted stock units (RSUs) to two new employees as inducement awards under the companys 2019 Inducement Plan.

Key Points: 
  • In addition, the company has granted an aggregate of 67,177 restricted stock units (RSUs) to two new employees as inducement awards under the companys 2019 Inducement Plan.
  • The grants were made in accordance with Nasdaq Listing Rule 5635(c)(4).
  • Vesting of the inducement grants is subject to continued service with AVROBIO by the employee through the applicable vesting dates.
  • AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.