Newsletter Update

Next-Generation Therapeutics | September Roundup

October 11, 2022
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Highlights & Summary

Intro

September was a month that saw a fair amount of regulatory activity, from BlueBird building on its Zynteglo success with Skysona, to Biomarin, Immusoft, and Sarepta progressing their respective assets through various regulatory stages and hurdles. Read below to find out more about these new key developments. 

Cell Therapy

  1. Immusoft brings a B cell therapy into the clinic for the first time | FDA 

  1. Scripps non-profit Calibr licenses “switchable” CAR-T platform to Abbvie | Partnerships

  1. Another step forward on the path towards solid tumor treatment | Clinical Trials

  1. A CD19 candidate stumbles as Servier ends a collaboration agreement with Allogene | Partnerships

  1. Capstan therapeutics joins the growing in vivo cell engineering field with $165M in funding | Funding

Gene Therapy

  1. After two-year delay, Biomarin resubmits BLA for Hemophilia A gene therapy | FDA

  1. Solid Bio and AavantiBio merge with additional funding from RA Capital and others | M&A

  1. Forge Biologics logs $90M series C to scale AAV production | Funding

  1. Bluebird has been on a roll with approvals | FDA

  1. Renovacor gets acquired by Rocket Pharmaceuticals as they look to expand their cardiac gene therapy profile | M&A

Oligo Therapy

  1. Orbital Therapeutics Launches to Advance New Frontiers of Science with the Next Generation of Innovative RNA Medicines | Company Creation

  1. Gatehouse Bio Extends Its Partnership with AstraZeneca to Develop RNA Therapeutics for Heart Failure | Partnership

  1. Alnylam and Regeneron Report Promising Data from Ongoing Phase 1 Study of ALN-HSD in NASH Patients and Healthy Volunteers | Clinical Trials

  1. Sarepta Therapeutics Announces That FDA has Lifted its Clinical Hold on SRP-5051 for Duchenne Muscular Dystrophy | FDA

  1. Lonza and Touchlight collaborate on end-to-end mRNA offering | Partnership

Cell Therapy

Immusoft brings a B cell therapy into the clinic for the first time | FDA

B Cell therapies have been drawing interest for a long time, but this is the first one to actually get into the clinic. The field is small compared to T cell therapies, but with a large amount of potential.

Scripps non-profit Calibr licenses “switchable” CAR-T platform to Abbvie | Partnerships

Calibr has been working towards a cell therapy for some time. They first showed clinical data at the end of 2021. The Abbvie collaboration gives them the opportunity to really push their platform forward and start bringing therapies to the clinic.

Another step forward on the path towards solid tumor treatment | Clinical Trials

Adaptimmune gave a data update on their Phase 1 trial using a CD8 TCR-T cell therapy. Solid tumors remain the golden goose in the field and Adaptimmune’s approach is also novel, since CD8 TCR-Ts have yet to find a place in the clinic.

A CD19 candidate stumbles as Servier ends a collaboration agreement with Allogene | Partnerships

CD19-targeting candidates already face an uphill battle as the space is very crowded. Allogene’s odds to pull off a win here just got quite a bit worse.

Capstan therapeutics joins the growing in vivo cell engineering field with $165M in funding | Funding

In vivo engineering for next-gen therapies is still open to all comers. Capstan already had some big names in their $63 million seed round led by Novartis Venture (themselves already big players in the space). This time Pfizer took the lead, although the cap table reads like a who’s who of life science VC.

Gene Therapy

After two-year delay, Biomarin resubmits BLA for Hemophilia A gene therapy | FDA

Biomarin has been patiently working to get back into the Hemophilia A gene therapy race in the U.S. They are already on the market in the EU and hopeful they have the data to move forward with U.S. sales as well.

Solid Bio and AavantiBio merge with additional funding from RA Capital and others | M&A

There has been a rash of M&A in the next-gen therapeutics space, with a lot coming from CDMO plays. Solid and AavantiBio are looking to capitalize on this by merging their two companies. The move sparked interest from investors as they received $75M in additional funding from Perceptive Advisors, RA Capital, and Bain Capital Life Sciences.

Forge Biologics logs $90M series C to scale AAV production | Funding

AAV CMC has been a hot topic and Forge has closed on additional funding to enhance its capacity. The funding will go towards not only improving capacity, but also getting ahead of regulatory requirements, which are still developing.

Bluebird has been on a roll with approvals | FDA

Bluebird went from zero to two approvals recently. First Zynteglo for beta-thalassemia, and now Skysona for cerebral adrenoleukodystrophy. Two diseases with high unmet-need. The approvals come after a string of setbacks for the company.

Renovacor gets acquired by Rocket Pharmaceuticals as they look to expand their cardiac gene therapy profile | M&A

Cardiac gene therapy is a popular topic and Rocket pharmaceuticals has decided to double down in the indication by acquiring Renovacor. The all-stock transaction has already been approved and should close in Q1 2023.

Oligo 

Orbital Therapeutics Launches to Advance New Frontiers of Science with the Next Generation of Innovative RNA Medicines | Company Creation

Orbital Therapeutics has launched with the goal of building a first-in-kind platform to advance a portfolio of RNA therapeutics across a range of human diseases. The company has also announced a collaboration with Beam Therapeutics to accelerate the development of a diverse range of assets.

Gatehouse Bio Extends Its Partnership with AstraZeneca to Develop RNA Therapeutics for Heart Failure | Partnership

Building on their existing partnership, Gatehouse Bio and AstraZeneca are advancing their partnership to create therapeutics for the treatment of HFpEF. Gatehouse Bio discovered mutations that were predicted to regulate biological pathways associated with the clinical syndrome.

Alnylam and Regeneron Report Promising Data from Ongoing Phase 1 Study of ALN-HSD in NASH Patients and Healthy Volunteers | Clinical Trials

Armed with powerful phase 1 data showing that a single dose of ALN-HSD was sufficient to induce a robust target knockdown and lower NAFLD activity score, a phase 2 is planned to start recruiting patients in Q4 of 2022.

Sarepta Therapeutics Announces That FDA has Lifted its Clinical Hold on SRP-5051 for the Treatment of Duchenne Muscular Dystrophy | FDA

A clinical hold on SRP-5051, caused by serious adverse events, was lifted after the FDA and Sarepta agreed on implementing changes to the protocol to resume dosing U.S patients. The MOMENTUM phase 2 trial is on track to finish enrolling patients by the end of 2022.

Lonza and Touchlight collaborate on end-to-end mRNA offering | Partnership

The Lonza - Touchlight collaboration enables Lonza to access Touchlight’s doggybone DNA platform (dbDNA) which would accelerate manufacturing and allow the company to offer an end-to-end solution for customers.

Authors
Rebecca Burnham
Associate Product Manager
Joe Daccache
Life Science Expert Level II
Luka Jelcic
Senior Associate
William Orent
Life Science Expert
Carl Schoellhammer
Principal
Kalyn Specht
Life Science Expert
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