Ciliopathy

ALSA Ventures launches novel Gene Therapy portfolio company Axovia Therapeutics to treat Ciliopathies

Retrieved on: 
Tuesday, September 19, 2023

LONDON, Sept. 19, 2023 (GLOBE NEWSWIRE) -- ALSA Ventures , a UK-based, therapeutics-focused venture capital fund, announces the acquisition of Axovia Therapeutics Inc and the launch of a new portfolio company Axovia Therapeutics Ltd .

Key Points: 
  • LONDON, Sept. 19, 2023 (GLOBE NEWSWIRE) -- ALSA Ventures , a UK-based, therapeutics-focused venture capital fund, announces the acquisition of Axovia Therapeutics Inc and the launch of a new portfolio company Axovia Therapeutics Ltd .
  • Axovia is developing the first gene therapies for ciliopathies and has a pipeline of products for these devastating diseases, including Bardet-Biedl Syndrome (BBS).
  • AXV101 is an AAV9-based gene therapy targeting retinal dystrophy associated with BBS in patients carrying biallelic mutations in the BBS1 gene.
  • Axovia Acting CEO Professor Phil Beales said the Axovia gene therapy platform gives hope to BBS patients worldwide.

ReCode Therapeutics to Participate in Upcoming August Investor Conferences

Retrieved on: 
Tuesday, August 3, 2021

ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced that Company leadership will host 1x1 meetings at two upcoming virtual investor conferences in August:

Key Points: 
  • ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced that Company leadership will host 1x1 meetings at two upcoming virtual investor conferences in August:
    ReCode Therapeutics is an integrated genetic medicines company developing disease-modifying therapeutics using powerful LNP delivery technology to target organs and tissues beyond the liver.
  • The Companys pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia.
  • The Company is leveraging its proprietary LNP platform and nucleic acid technologies and utilizing systemic and direct delivery for mRNA-mediated replacement and gene editing/correction in target cells, including stem cells.
  • For more information, visit www.recodetx.com and follow us on Twitter @ ReCodeTx and LinkedIn .

ReCode Therapeutics to Present Preclinical Data from mRNA-Based Program for Primary Ciliary Dyskinesia at the 2021 PCD on the Move Virtual Scientific Conference

Retrieved on: 
Friday, July 30, 2021

Archived replays of each presentation will be made available on the Companys website following the presentations.

Key Points: 
  • Archived replays of each presentation will be made available on the Companys website following the presentations.
  • ReCode Therapeutics is an integrated genetic medicines company developing disease-modifying therapeutics using powerful LNP delivery technology to target organs and tissues beyond the liver.
  • The Companys pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia.
  • For more information, visit www.recodetx.com and follow us on Twitter @ ReCodeTx and LinkedIn .

Rhythm Pharmaceuticals Announces Collaborative Research Agreement with Clinical Registry Investigating Bardet-Biedl Syndrome

Retrieved on: 
Wednesday, July 28, 2021

BOSTON, July 28, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, today announced a collaborative research agreement with the Clinical Registry Investigating Bardet-Biedl Syndrome (CRIBBS).

Key Points: 
  • BOSTON, July 28, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company committed to transforming the care of people living with rare genetic diseases of obesity, today announced a collaborative research agreement with the Clinical Registry Investigating Bardet-Biedl Syndrome (CRIBBS).
  • The registry is designed to advance the understanding of BBS and its impact on patients and families, improve the care of people living with the disease, and augment scientific and clinical research.
  • It is supported by the BBS Foundation and Family Association, Bright Light to BBS, and Marshfield Clinic Research Institute.
  • Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity.

ReCode Therapeutics to Present at William Blair Biotech Focus Conference 2021

Retrieved on: 
Thursday, July 8, 2021

ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its SORT-LNPTM platform, today announced that CEO and President, David Lockhart, Ph.D., will participate in a panel discussion on non-viral drug delivery at the upcoming William Blair Biotech Focus Conference 2021 at 2:00 pm Eastern Time on Thursday, July 15, 2021.

Key Points: 
  • ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its SORT-LNPTM platform, today announced that CEO and President, David Lockhart, Ph.D., will participate in a panel discussion on non-viral drug delivery at the upcoming William Blair Biotech Focus Conference 2021 at 2:00 pm Eastern Time on Thursday, July 15, 2021.
  • An archived replay of the presentation will be made available on the Companys website following the presentation.
  • ReCode Therapeutics is an integrated genetic medicines company developing disease-modifying therapeutics using its powerful, proprietary SORT-LNPTM platform to target organs and tissues beyond the liver.
  • The Companys pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia.

ReCode Therapeutics to Participate in Upcoming June Investor Conferences

Retrieved on: 
Monday, June 14, 2021

ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its SORT-LNP platform for the treatment of life-limiting respiratory diseases, today announced that the Company will participate in two upcoming virtual investor conferences in June:

Key Points: 
  • ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its SORT-LNP platform for the treatment of life-limiting respiratory diseases, today announced that the Company will participate in two upcoming virtual investor conferences in June:
    Format: Presentation and Q&A by David Lockhart, Ph.D., CEO and President
    An archived replay of the presentation will be made available on the Companys website following the presentation.
  • ReCode Therapeutics is an integrated genetic medicines company developing disease-modifying therapeutics using its powerful, proprietary SORT-LNP platform to target organs and tissues beyond the liver.
  • The Companys pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia.
  • For more information, visit www.recodetx.com and follow us on Twitter @ ReCodeTx and LinkedIn .

ReCode Therapeutics to Present Preclinical Data Using SORT-LNP™ and RNA Platforms to Rescue CFTR Function at the 44th European Cystic Fibrosis Conference (ECFS)

Retrieved on: 
Monday, May 24, 2021

Date and Time: Friday, June 11, 2021, 9:00 - 10:00 a.m. CET (3:00 - 4:00 a.m. EST)

Key Points: 
  • Date and Time: Friday, June 11, 2021, 9:00 - 10:00 a.m. CET (3:00 - 4:00 a.m. EST)
    The abstract will be available on the ECFS website beginning on Wednesday, June 2, 2021.
  • The presentation will be made available on the ECFS website for viewing at the start of the presentation on June 11, 2021 at 9:00 a.m. CET (3:00 a.m. EST).
  • ReCode Therapeutics is an integrated genetic medicines company developing targeted, disease-modifying therapeutics using its powerful, proprietary SORT-LNPTM platform to target organs and tissues beyond the liver.
  • The Companys pipeline includes lead programs for patients with life limiting respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia.

Albireo Announces First Patients Dosed in Two New Studies

Retrieved on: 
Thursday, March 25, 2021

Additionally, the Company enrolled its first patient in the ASSERT Study, a global Phase 3 pivotal trial of odevixibat in patients with Alagille syndrome (ALGS).

Key Points: 
  • Additionally, the Company enrolled its first patient in the ASSERT Study, a global Phase 3 pivotal trial of odevixibat in patients with Alagille syndrome (ALGS).
  • I am proud of the work and great progress we are making in our studies of odevixibat in rare cholestatic diseases, said Patrick Horn, Chief Medical Officer of Albireo.
  • An additional exploratory cohort of patients 18 years of age with genetically confirmed diagnosis will be enrolled, not to exceed 18 patients in total.
  • As a result of risks and uncertainties that Albireo faces, the results or events indicated by any forward-looking statement may not occur.

Renowned Geneticist Philip Beales Launches Axovia Therapeutics to Develop Transformative Medicines for Ciliopathies

Retrieved on: 
Thursday, February 25, 2021

Axovia is a majority-owned subsidiary of Jaguar Gene Therapy, which is backed by Deerfield Management, and is also partially funded by UCL Technology Fund .

Key Points: 
  • Axovia is a majority-owned subsidiary of Jaguar Gene Therapy, which is backed by Deerfield Management, and is also partially funded by UCL Technology Fund .
  • Our goal is to build on the scientific discoveries and molecular diagnosis of Bardet-Biedl syndrome to accelerate the development of disease-transformative treatments for patients as early as possible, said Philip Beales, CEO and co-founder of Axovia Therapeutics.
  • The Axovia team is currently working to develop a gene therapy treatment for BBS1, a subset of BBS, which is life-threatening and affects many organs of the body.
  • We at Jaguar are excited by the vision of Professor Beales and the Axovia team to develop transformative therapies for patients suffering from ciliopathies, said Joe Nolan, Chief Executive Officer of Jaguar Gene Therapy.

Eloxx Announces Publication of Scientific Manuscript on ELX-02 in the Journal of Cystic Fibrosis

Retrieved on: 
Tuesday, February 9, 2021

These data support our current Phase 2 clinical trial for ELX-02 in cystic fibrosis patients with G542X nonsense alleles, for whom there are few, if any, treatment options available.

Key Points: 
  • These data support our current Phase 2 clinical trial for ELX-02 in cystic fibrosis patients with G542X nonsense alleles, for whom there are few, if any, treatment options available.
  • ELX-02 is in the early stages of clinical development focusing on cystic fibrosis.
  • Eloxx also has preclinical programs focused on kidney diseases including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.
  • Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control.