1. Scrip
  2. >>Business

Avrobio Plots Comeback With Early Data From Gaucher’s Gene Therapy

Sees Bluebird Success As Creating Pathway

 
• By Andrew McConaghie

Early data in Gaucher disease suggest Avrobio’s hematopoietic stem cell gene therapy could help patients who do not response to enzyme replacement therapy.

Avrobio
Avrobio is looking to revive its fortunes by developing a Gaucher's gene therapy. • Source: Shutterstock

Avrobio is looking to bounce back from an earlier failure with its hematopoietic stem cell (HSC) gene therapy platform, and has just unveiled promising initial data from the first five patients treated for Gaucher’s disease with its investigational candidate, AVR-RD-02.

Gaucher’s disease is the most common lysosomal disorder, affecting 50,000 to 100,000 people worldwide. Characterized by a build up of...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Business

Sarepta Sells Arrowhead Shares To Raise Cash, Pay Arrowhead

 
• By Mandy Jackson

Sarepta is in a cash crunch with sales of Elevidys slumping due to safety concerns and with financial commitments coming due, including debt repayments and milestone fees for its partner Arrowhead.

Deal Watch: Lilly Teams With GPCR-Focused Superluminal In Cardiometabolic Disease

 
• By Joseph Haas

Plus deals involving Basilea/Venatorx, CorMedix/Melinta, SERB/Y-mAbs, XOMA/Lava, XOMA/Hillevax, MaxCyte/Adicet/Anocca and Bausch Health/DURECT.

Schrödinger Drops CDC7 Inhibitor SGR-2921 After Two Patient Deaths

 
• By Alaric DeArment

The drug is one of multiple candidates in the class that have been pulled from development, though it is not the company’s lead candidate.

Finance Watch: OrbiMed’s New $1.86bn Fund Offers Financial Alternatives In Tough Market

 
• By Mandy Jackson

Public Company Edition: OrbiMed partners Carter Neild and Matthew Rizzo spoke with Scrip about the firm’s fifth royalty and credit fund. Also, Ligand sold $400m worth of notes, BridgeBio’s oncology spinout went public via SPAC merger and raised $261m and Assembly raised $175m.

More from Scrip

Vedanta’s Live Bacteria Reach A Dead End In Ulcerative Colitis

 
• By Andrew McConaghie

The microbiome-based company’s trial of VE202 in ulcerative colitis has failed in Phase II, but its ongoing Phase III study in C difficile infection is more promising.

Finance Watch: OrbiMed’s New $1.86bn Fund Offers Financial Alternatives In Tough Market

 
• By Mandy Jackson

Public Company Edition: OrbiMed partners Carter Neild and Matthew Rizzo spoke with Scrip about the firm’s fifth royalty and credit fund. Also, Ligand sold $400m worth of notes, BridgeBio’s oncology spinout went public via SPAC merger and raised $261m and Assembly raised $175m.

Asia Deal Watch: Lupin Picks Sandoz To Market Lucentis Biosimilar In Parts Of EU And Asia

 
• By Joseph Haas, Sushmita Panda, Xu Hu, Vibha Ravi, and Manas Mishra

Also deals involving Xspray/Handa, Fosun/Expedition, Kaken/Astria, Orchid/Allecra, Sanofi/Visirna, KKR/Healthcare Royalty Partners, Viridian/Kissei, Sandoz/Evotec, Dx&Vx, Dr. Falk/Allianthera, Madrigal/CSPC and Lilly/LTZ.