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Next-Generation Therapeutics Newsletter | June Roundup

June 29, 2022
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Highlights & Summary

Intro

June was a busy month with funding rounds, study results, and manufacturing partnerships bringing a range of next gen therapeutics closer to the clinic. Happy reading!

Cell Therapy

  1. New insights into the CAR-T cytokine release syndrome from a Medidata study  | Research
  1. Adicet moves one step closer to a gamma-delta CAR-T therapeutic | Clinical Trials
  1. Another step forward in “off-the-shelf” cell therapies with Precision’s Phase 1/2 results | Clinical Trials
  1. Tessa Therapeutics secures instrumental $126M to move CAR-T candidate into pivotal trial | Financing
  1. MD Anderson and Resilience team up on a 60,000 sqft cell therapy manufacturing center | Manufacturing

Gene Therapy

  1. Interim readout shows uniQure’s Huntington’s Disease candidate is well tolerated | Clinical Trials 
  1. Stealth China-U.S. company takes in $160M Series B lead by OrbiMed | Financing
  1. Despite past setbacks, Astellas opens new AAV manufacturing facility | Manufacturing
  1. BioMarin’s ROCTAVIAN receives recommendation for approval from EMA | Regulatory
  1. LogicBio solidifies manufacturing supply with partnerships | Partnership 

Oligo Therapy
 

  1. FDA places a clinical hold on Sarepta’s SRP-5051 Duchenne Muscular Dystrophy treatment | Regulatory
  1. Alnylam scores FDA approval of AMVUTTRA™ (vutrisiran), a RNAi rare disease drug | Regulatory
  1. Intellia and Regeneron’s phase 1 data show CRISPR-based drug, led to durable TTR reductions in ATTR | Clinical Trials
  1. Phase 2 Fazirsiran data shows improvement in liver health biomarkers Antitrypsin Deficiency | Clinical Trials
  1. Code Biotherapeutics raises upsized and oversubscribed $75 million in Series A | Financing

Cell Therapy

  1. New insights into the CAR-T cytokine release syndrome from a Medidata study  | Research

Medidate used its Acorn AI platform to examine pooled data from 542 patients and claims to have identified a set of commonly measured biomarkers which predict cytokine release syndrome (CRS). CRS is one of the main risks with CAR-T therapies and is a common source of concern in determining optimal CAR-T dosing.

  1. Adicet moves one step closer to a gamma-delta CAR-T therapeutic | Clinical Trials

Gamma-delta T cells are interesting therapeutic candidates because of their high cytotoxicity and potential for off-the-shelf use. Adicet released very promising Phase 1 data, including favorable response rates in the higher doses.  

  1. Another step forward in “off-the-shelf” cell therapies with Precision’s Phase 1/2 results | Clinical Trials

Allogeneic CAR-T therapies are still a goal in the field, and the race is on for the first real viable product. Precision Biosciences offered positive interim data with their ongoing Phase 1/2 study which is estimated to be completed in Q2 2024.

  1. Tessa Therapeutics secures instrumental $126M to move CAR-T candidate into pivotal trial | Financing

The Singaporean biotech has both autologous and allogeneic anti-CD30 CAR-T candidates in development. CD30 is a strong lymphoma target but there are no approved cellular therapies against it. Tessa has promising Phase 1 and 2 data and securing this funding will support their pivotal trial with the autologous candidate.

  1. MD Anderson and Resilience team up on a 60,000 sqft cell therapy manufacturing center | Manufacturing

The Cell Therapy Manufacturing Center is a novel project meant to accelerate both the development and production of a variety of cellular therapies. Since manufacturing has been a persistent thorn in the side of cellular therapies there has been great interest in novel strategies to produce these products for the clinic.

Gene Therapy

  1. Interim readout shows uniQure’s Huntington’s Disease candidate is well tolerated | Clinical Trials 

uniQure’s interim readout from a phase 1/2 trial testing their candidate for Huntington’s disease showed the therapy to be well tolerated and reduces mutant huntingtin protein, a key biomarker.

  1. Stealth China-U.S. company takes in $160M Series B lead by OrbiMed | Financing

OrbiMed led a Series B for a stealth, China-U.S. company, Frontera Therapeutics, which is planning clinical trials for their lead program addressing a rare, undisclosed eye disease. The company has a GMP manufacturing facility in Suzhou, China.

  1. Despite past setbacks, Astellas opens new AAV manufacturing facility | Manufacturing

Astellas completes a new 135,000 sq.ft. manufacturing facility to support their pipeline of AAV therapies. The facility provides Astellas with 4,000 liters of production capacity, with the capability to expand and accommodate up to 16,000 liters in the future.

  1. BioMarin’s ROCTAVIAN receives recommendation for approval from EMA | Regulatory

BioMarin’s Roctavian (valoctocogene roxaparvovec), a gene therapy for Hemophilia A,  received conditional approval from the EMA. Roctavian is the first gene therapy to be recommended for approval in Europe for hemophilia A. 

  1. LogicBio solidifies manufacturing supply with partnerships | Partnership 

LogicBio partners with Exothera SA and Polyplus to create a scalable AAV production platform, with a capacity of 200 - 2,000L using Pall’s Allegro single-use bioreactors

Oligo Therapy

  1. FDA places a clinical hold on Sarepta’s SRP-5051 Duchenne Muscular Dystrophy treatment | Regulatory

The FDA halted part B of the Duchenne Muscular Dystrophy (DMD) Phase 2 study for SRP-5051 (vesleteplirsen), following a serious low magnesium level adverse event. Sarepta is still on track to finish enrolling patients by the end of the year and will provide the FDA with all safety information expeditiously, including non-serious cases. SRP-5051 is the most advanced asset in Sarepta’s pipeline.

  1. Alnylam scores FDA approval of AMVUTTRA™ (vutrisiran), an RNAi rare disease drug  | Regulatory

Alnylam was granted approval for its second hAATR asset after it had met primary and all secondary endpoints. Company expects to launch in early July, with Value-Based Agreements to accelerate access. This is building on Alnylam’s previous successes in this indication having launched Onpattro’s four years ago.

  1. Intellia and Regeneron’s phase 1 data show CRISPR-based drug, led to durable TTR reductions in ATTR| Clinical Trial

The much-anticipated Phase 1 data for the first in vivo CRISPR-gene editing program (NTLA-2201), show sustained reduced levels of TTR in ATTR patients. NTLA-2001 is also being investigated as a single dose therapy for cardiomyopathy (ATTRwt). 

  1. Phase 2 Fazirsiran data shows improvement in liver health biomarkers Antitrypsin Deficiency | Clinical Trials

Arrowhead and Takeda announced results of the Phase 2 study in alpha-1 antitrypsin deficiency (AATD), showing substantial reduction in Fibrosis, histological burden, as well as Z-AAT accumulation in the liver. There is currently no treatment for AATD-driven liver disease. Vertex has previously been unsuccessful in this space.

  1. Code Biotherapeutics raises upsized and oversubscribed $75 million in Series A | Financing

Code Bio has closed a substantial Series A financing round to tackle both Duchenne Muscular Dystrophy and Type 1 Diabetes. CodeBio will be relying on its 3DNA® platform that aims to take on viral vectors.

Authors
Michelle Atallah
Life Science Expert
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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