10% RIF at Encoded Therapeutics. ETX101 (SCN1A AAV gtx for Dravet) Phase 1/2 still not recruiting despite originally being posted in June ‘22

RIF of undisclosed size at Xalud Therapeutics, private non-viral pDNA gene therapy company. Discontinuing OA, pivoting to ALS

RIF of undisclosed size at Rejuvenate Bio, aging-focus AAV gene therapy company Raised a $14.5M Series A in 2021

Perhaps not so surprising anymore!

Value creation to value realization $NVS to acquire $KDNY for up to $3.5B. $3.2B upfront (67% premium) + CVR

Pharma interest in Treg cell therapy still going strong $AZN / Quell to collaborate on CAR-Tregs for IBD and T1D. $85M upfront, up to $2B potential biobucks

Interesting to see FDA grant $CRSP / $VRTX different review timelines for SCD and B-Thal (reflecting ZYNTEGLO availability) PDUFAs: SCD - 12/8/23 B-Thal - 3/30/24

Pretty remarkable that $BMY's ratio of CAR-T manufacturing employees to treated patients is 1:1 6 facilities and 4K employees for $677M in LTM revenue. Cell therapy is hard!

Kate deal gives $ALPMY full O/S of a next-gen XLMTM asset We know $BOLD showed meaningful efficacy but was hampered by tox. Presumably, thesis is Kate's novel myotropic capsid (MyoAAV) will enable equivalent efficacy at lower doses with less tox Financials undisclosed

$ALPMY taking an asset-centric, bolt-on approach to bolster its AAV pipeline First, $TSHA deal for TSHA-120 (GAN) & TSHA-102 (Rett) Now, Kate deal for KT430 (XLMTM) Despite $BOLD's failures, encouraging to see continued investment in the wake of others' exits (PFE, TAK)

Fascinating biology. Spontaneous ATTR-CM regression and cardiac remodeling driven by polyclonal anti-amyloid IgG response. Seen in 3 pts of 1,663 cases Cool “real world” proof of mechanism for anti-TTR mAbs $AZN $NVO $PRTA

*Sigh*

A lot of denominator gymnastics with $ALLO, but these data do show that long-term, durable, multi-year CRs *are* possible (1) with allogeneic cells; and (2) in the absence of persistence

Nice head-to-head comp vs. Tecartus here, including real-world data from ROCCO. Obe Cel durability to be updated at ASH $AUTL $GILD

$AUTL CR/CRi 76%***

$AUTL Obe Cel vs $GILD Tecartus pivotal Adult ALL data H2H: CRi: 71% vs 65% (both 97% MRD-) mDOR: NR (61% @ 9.5M) vs 13.6M Gr3+ CRS: 3% vs 26% Gr3+ ICANS: 7% vs 35% Should be interesting to see how much safety matters commercially vs. entrenched incumbent w/ broader label &…

Congratulations @bradloncar! An exciting new addition to the biotech ecosystem. Brad’s interviews are always value-add, a product of his willingness to ask the tough questions. Looking forward to seeing him scale these efforts and further democratize access to industry leaders

$3.5-4B+ post for a TLR agonist and a PARP? Quite the Perlmutter Premium

Experimenting w/ Blue. Looks like editing the 1st tweet in a thread deletes all subsequent tweets... Other "1H / Mid-23 Events": $STOK 45mg Dravet $ROIV TL1a Maintenance $AMGN TSLP Ph2 CSU $RARE Setrusumab Ph2 $FIXX PKU Ph1/2 $AGIO SCD Go/No-Go $VTYX TKY2 ER Formulation

Upcoming events and expected moves from Cowen. Should be an exciting June to wrap up Q2. Some confirmed events: $MLTX IL-17 Ph2 data $QURE & $PTCT HD data $ACET CD20 gdCAR-T update $FDMT CF functional data High-profile PDUFAs ($BMRN Roctavian, $SRPT SRP-9001)

Upcoming events and expected moves from Cowen. Should be an exciting June to wrap up Q2. Some confirmed events: $MLTX IL-17 Ph2 data $QURE & $PTCT HD data $ACET CD20 gdCAR-T update $FDMT CF functional data High-profile PDUFAs ($BMRN Roctavian, $REGN HD Eylea, $SRPT SRP-9001)

Disappointingly little PK/PD in $DTIL PBCAR19B data, which was industry's first look at B2M KO + HLA-E KI allo construct Why is T/NK reconstitution data N=2 if 7 pts. were treated? Where are CAR+ cell exposure / AUC curves? Unfortunately, largely uninterpretable & inconclusive

RA-backed non-viral gene therapy company Summation Bio is shutting down, per Endpoints ~$25M of total $65M Series A invested per 2022 Reg D filing

Putting that all together for $AVRO: $1M upfront license payment $16.5M direct R&D spend $10M sub-license income share ~$27.5M spent, $87.5M sale, $60M net In ~6 years, yielding a 57% IRR Not too shabby! (6/6)

In addition to its downstream economics, Papillon was entitled to a low double digit share of sub-license income If we assume 11-12%, Papillon (and Cherqui) received a nice payday from $NVS, to the tune of ~$10M (5/6)