We head into the historically quiet 4th July week off the back of another booming week of good news in biotech. Money continues to move towards immunology, propping up the sector with deals and raises. There were some huge developments that cannot be understated, such as patients being safety dosed with gene editing drugs for the first time ever, the potential for a one and done GLP gene therapy to treat obesity, and a novel approach to gene editing that could best all that have come before it. In a time when the sector has defaulted away from novel modalities towards "safer bets", there has been a good amount of activity in the RNA, cell therapy, gene therapy and gene editing spaces. Enjoy!
🚀 New Biotech Launches & Private raises…
-
Formation Bio
secures a mega $372M series D and big pharma backing to license 10 new programs and expand its AI Development capabilities Link
- Biotech VC disruptor
Curie.Bio
boosts its fund by $380M, bringing total funds to almost $1B as it to continue to invest in biotechs on a path to generate meaningful clinical data Link
- In a continued trend of launching clinical ready companies,
Ensho Therapeutics
emerges with a seasoned leadership team Phase 2 ready oral α4β7 inhibitor for Inflammatory Bowel Disease acquired from
Eisai US
Link
-
Exsilio Therapeutics
launched with an $82M Series A and ex-Moderna Exec Tal Zaks as interim-CEO to develop genomic medicines using naturally occurring, programmable genetic elements that can insert new genes into the cell through mRNA intermediates Link
🤝 Deals deals deals…
-
AbbVie
acquires immunology biotech
Celsius Therapeutics
for $250M in a deal centered around a phase 1 potentially first-in-class anti-triggering receptor expressed on myeloid cells 1 (TREM1) antibody for treating inflammatory bowel disease. Link
-
Novo Nordisk
purchased
2seventy bio
's hemophilia A program along with rights to some of the biotech’s gene editing tech for autoimmune diseases in a deal worth up to $40 million. Link
-
Eli lilly
announced a new collaboration with
OpenAI
to discover novel medicines to treat drug-resistant bacteria Link
- Searching for a path forward after the withdrawal of Relyvrio in amyotrophic lateral sclerosis (ALS),
Amylyx Pharmaceuticals
has purchased a GLP-1 receptor antagonist from
Eiger BioPharmaceuticals
for $35.1 million. Link
- Despite broader business challenges, early AI drug discovery pioneer
BenevolentAI
expanded their collaboration with
AstraZeneca
to include a novel lupus target. Another sign on the hot immunology market. Link
-
Dewpoint Therapeutics
strengthened it collaboration with
Novo Nordisk
to leverage its HTS genome-wide CRISPR screening platform with Novo's target discovery engine Link
📰 IPO News…
- Immunology biotech
Alumis
announced final pricing of its IPO however did downsize from 17.6 million shares at $17 per share ($299M) 13.1 million at $16 ($209M) and included a private placement from existing investor AyurMaya Capital Management. The IPO window remains open but is still a challenging environment... Link
📈 Positive clinical development updates…
- In what could be a defining moment for the future of gene editing and in a world first,
Intellia Therapeutics, Inc.
announced can safety dose patients with its CRISPR gene editing therapy more than once, even though it does not need to Link
- Another very closely watched trial is
Alnylam Pharmaceuticals
's RNAi HELIOS-B Phase 3 Study of Vutrisiran, which presented positive topline results, claiming to hit all primary and secondary endpoints in a heart disease trial Link
-
Wave Life Sciences
announced positive results from a Phase 1b/2a trial for its ASO therapeutic treating Huntington’s Disease. Opening the door for potential accelerated approval and an opt-in from partner
Takeda
Link
-
Carisma Therapeutics
bounce back from a major trial failure earlier this year as the FDA grant fast track designation for its HER2 targeting ex-vivo gene-modified autologous chimeric antigen receptor-monocyte cell therapy to treat solid tumors Link. The biotech also announced nomination of a first In-vivo CAR-M for Hepatocellular Carcinoma in collaboration with
Moderna
. Link
-
Beam Therapeutics
announced that it has dosed the first patient in the highly anticipated Phase 1/2 study of BEAM-302 to treat Alpha-1 Antitrypsin Deficiency (AATD) Link
-
ReCode Therapeutics
receive US FDA Orphan Drug Designation for its inhaled mRNA-LNP drug to treat primary ciliary dyskinesia (PCD), which currently has no approved treatments Link
-
Johnson & Johnson
presented phase 3 data on nipocalimab for the treatment of rare autoimmune disease generalized myasthenia gravis, providing insight into how the prospect it acquired in its $6.5B acquisition of Momenta Pharmaceuticals in 2020 will compete with rival programs from
argenx
and
UCB
. Link
- In the 2nd inhaled mRNA drug news of the week,
Ethris
announced interim phase 1 data to show that its lead candidate appears safe and increases expression of a key protein to prevent viral infections in people with uncontrolled asthma. Link
-
Silence Therapeutics plc
announced positive results of its phase 1 siRNA program treating Polycythemia Vera patients. Link
-
CalciMedica
's calcium channel inhibitor helps a subgroup of pancreatitis patients eat solids sooner in a phase 2 trial. Link
-
Takeda
presented positive phase 2 data for mezagitamab, which increased platlet count in patients with Primary Immune Thrombocytopenia. Link
-
Novo Nordisk
shared more phase 3 data on its hemophilia A candidate Mim8, revealing new data that show how it squares up against
Roche
's blockbuster Hemlibra on key measures of efficacy and tolerability. Link
🐁 Preclinical obesity news…
-
Terns Pharmaceuticals
MASH drug was shown preclinically to not only reduced weight, but also retain lean muscle mass, when used in combination with Novo's semaglutide. This could continue to raise the bar for emerging obesity treatments! Link
-
Fractyl Health
are addressing the problem of weight gain returning after a patient stops taking GLP-1 drug with an AAV gene therapy that integrates a GLP-1-secreting transgene directly into the beta islet cells of the pancreas, locking in the mechanism that gives weight loss drugs their benefits. Definitely a space to watch as a one and done obesity drug would be a game changer! Link
🧬 Cool science of the week…
Last but definitely not least in terms of updates from last week...
Researchers from the
Arc Institute
had their mic drop moment last week as the culmination of years of work resulted in 2 Nature papers describing a novel approach to genome editing involving "bridge RNAs". These RNAs bind specifically to their encoded recombinase and contain two internal loops that can be independently reprogrammed. By directing sequence-specific recombination between DNA molecules, this modularity enables DNA insertion, excision, and inversion. Unlike CRISPR or RNA interference, bridge recombination works in vitro without relying on cellular repair mechanisms, potentially leading to safer genome edits.
Good luck bringing this research further
Matthew Durrant
,
Nicholas Perry
and team!
That's all for this week. Subscribe for alerts to the latest positive news in the industry ⬇️🔔
About the author: Max Robinson is an ex-scientist turned talent leader in the biotechnology industry, and currently Global Head of Discovery & Preclinical Research at Proclinical.
Max Robinson - excellent newsletter!